Ignite Creation Date:
2025-12-25 @ 12:36 AM
Ignite Modification Date:
2025-12-31 @ 12:40 PM
Study NCT ID:
NCT02652767
Status:
COMPLETED
Last Update Posted:
2022-07-29
First Post:
2016-01-07
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D029242', 'term': 'Optic Atrophy, Hereditary, Leber'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D028361', 'term': 'Mitochondrial Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}], 'ancestors': [{'id': 'D015418', 'term': 'Optic Atrophies, Hereditary'}, {'id': 'D009896', 'term': 'Optic Atrophy'}, {'id': 'D009901', 'term': 'Optic Nerve Diseases'}, {'id': 'D003389', 'term': 'Cranial Nerve Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'mtaiel@gensight-biologics.com', 'phone': '1 76 21 72 26', 'title': 'Magali Taiel CMO', 'phoneExt': '0033', 'organization': 'GenSight Biologics'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first dose to end of study (a maximum of 96 weeks)', 'description': 'Safety population: All participants who were randomized and received study treatments.\n\nBecause all participants received both study treatment and the sham procedure simultaneously, adverse events (AEs) are reported overall for systemic and ocular events. Reported events include AEs associated with both GS010 and the sham procedure.', 'eventGroups': [{'id': 'EG000', 'title': 'All Participants', 'description': 'All participants who were enrolled and received both study treatments, GS010 and Sham. All participants in the study received both GS010 and the sham procedure simultaneously. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham procedure in the eye not assigned to receive GS010, at the same study visit.\n\nGS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia.\n\nSham: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.', 'otherNumAtRisk': 39, 'deathsNumAtRisk': 39, 'otherNumAffected': 38, 'seriousNumAtRisk': 39, 'deathsNumAffected': 2, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Blood glucose increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Neutrophil count increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'White blood cell count increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 6}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Alcohol use', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Social circumstances', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Anterior chamber cell', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 6}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Anterior chamber flare', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 5}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Anterior chamber inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Autoimmune uveitis', 'notes': 'The verbatim term for all events of "autoimmune uveitis" is "intermediate uveitis".', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 15}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Chalazion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Conjunctival haemorrhage', 'notes': '1/7 affected participants experienced this event in the sham-treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 7}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Conjunctival hyperaemia', 'notes': '4/5 affected participants experienced this event in the sham-treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 5}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Corneal disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Eye irritation', 'notes': '1/2 affected participants experienced this event in the sham-treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Eye pain', 'notes': '1/3 affected participants experienced this event in the sham treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Foreign body sensation in eyes', 'notes': '1/2 affected participants experienced this event in the sham treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Iridocyclitis', 'notes': '2/18 affected participants experienced this event in the sham-treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 18}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Iris atrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Iris transillumination defect', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Keratic precipitates', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 9}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Macular oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Photopsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Punctate keratitis', 'notes': '3/18 affected participants experienced this event in the sham-treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 18}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Vitreal cells', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Vitreous disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Vitreous floaters', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Vitritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 7}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Intraocular pressure increased', 'notes': '1/13 affected participants experienced this event in the sham-treated eyes.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 13}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}], 'seriousEvents': [{'term': 'Alcoholic liver disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Alcohol poisoning', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Malnutrition', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Alcohol withdrawal syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Renal failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.380', 'spread': '0.129', 'groupId': 'OG000'}, {'value': '0.392', 'spread': '0.129', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.8890', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.012', 'ciLowerLimit': '-0.182', 'ciUpperLimit': '0.158', 'groupDescription': 'A mixed model of analysis of covariance (ANCOVA) was used with change from baseline at Week 48 as the response, and participants, eyes of the participant as random factor, treatment and baseline LogMAR value as covariates in the model. P-value is used to assess the significance of the difference between All-GS010 and All-Sham with respect to change of LogMAR from baseline.', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline and Week 48', 'description': 'Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Visual acuity is measured in "logarithm of the minimal angle of resolution" (LogMAR), which was derived from the number of letters participants could read on the ETDRS chart.\n\n1 ETDRS line = 5 letters\n\n1 ETDRS line = 0.1 LogMAR\n\nA lower LogMAR score denotes better visual acuity. A positive change from baseline indicates a worsening in symptoms.\n\nChange = (Week 48 score - Baseline score).', 'unitOfMeasure': 'LogMAR', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in ETDRS Visual Acuity (Quantitative Score)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 72', 'categories': [{'measurements': [{'value': '0.192', 'spread': '0.104', 'groupId': 'OG000'}, {'value': '0.216', 'spread': '0.104', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'categories': [{'measurements': [{'value': '0.178', 'spread': '0.120', 'groupId': 'OG000'}, {'value': '0.207', 'spread': '0.120', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline; Week 72 and Week 96', 'description': 'Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Visual acuity is measured in "logarithm of the minimal angle of resolution" (LogMAR), which was derived from the number of letters participants could read on the ETDRS chart.\n\n1 ETDRS line = 5 letters\n\n1 ETDRS line = 0.1 LogMAR\n\nA lower LogMAR score denotes better visual acuity. A positive change from baseline indicates a worsening in symptoms.\n\nChange = (Week 72 score - Baseline score) or (Week 96 score - Baseline score). Missing data was imputed by the linear interpolation method.', 'unitOfMeasure': 'LogMAR', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Number of Eye Responders to Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48 Definition 1', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'Week 48 Definition 2', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}]}, {'title': 'Week 72 Definition 1', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}]}, {'title': 'Week 72 Definition 2', 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}, {'title': 'Week 96 Definition 1', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}]}, {'title': 'Week 96 Definition 2', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_UNITS', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'An eye was determined as a responder to treatment based on 2 different definitions.\n\nDefinition 1: An eye responder was defined by an improvement of the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity score of at least 15 letters compared to Baseline, or a final visual acuity greater than a Snellen acuity equivalent of 20/200 (a score of at least 1 letter).\n\nDefinition 2: An eye responder was defined by an improvement of the ETDRS score of at least 20 letters compared to Baseline.', 'unitOfMeasure': 'Eyes', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Number of Subject Responders to Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'All Participants', 'description': 'All participants who were enrolled and received both study treatments, GS010 and Sham. All participants in the study received both GS010 and the sham procedure. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham procedure in the eye not assigned to receive GS010, at the same study visit.\n\nGS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia.\n\nSham: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.'}], 'classes': [{'title': 'Week 48', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Week 72', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Week 96', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Week 48; Week 72 and Week 96', 'description': 'A subject responder was defined as a participant whose Early Treatment Diabetic Retinopathy Study (ETDRS) score of the treated eye (that received GS010), was at least 15 letters better than the sham eye, or whose treated eye had a "logarithm of the minimal angle of resolution" (LogMAR) acuity score of at least 0.3 LogMAR better than the sham eye.\n\nFor the Week 96 analysis, if no score was available for Week 96, the score from the previous visit was used.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-to-treat (ITT) population: All participants that received study treatments, with data at the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in GCL Macular Volume', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.184', 'spread': '0.014', 'groupId': 'OG000'}, {'value': '-0.207', 'spread': '0.014', 'groupId': 'OG001'}]}]}, {'title': 'Week 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.204', 'spread': '0.015', 'groupId': 'OG000'}, {'value': '-0.226', 'spread': '0.015', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.208', 'spread': '0.015', 'groupId': 'OG000'}, {'value': '-0.221', 'spread': '0.015', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Ganglion cell layer (GCL) macular volume was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).', 'unitOfMeasure': 'mm^3', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent -to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in RNFL Temporal Quadrant Thickness', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-22.8', 'spread': '1.0', 'groupId': 'OG000'}, {'value': '-24.7', 'spread': '1.0', 'groupId': 'OG001'}]}]}, {'title': 'Week 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-25.5', 'spread': '0.9', 'groupId': 'OG000'}, {'value': '-26.0', 'spread': '0.9', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '32', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-24.2', 'spread': '0.8', 'groupId': 'OG000'}, {'value': '-26.1', 'spread': '0.8', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Retinal nerve fiber layer (RNFL) temporal quadrant thickness was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).', 'unitOfMeasure': 'µm', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in RNFL Papillomacular Bundle Thickness', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-10.4', 'spread': '1.0', 'groupId': 'OG000'}, {'value': '-12.4', 'spread': '1.0', 'groupId': 'OG001'}]}]}, {'title': 'Week 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-12.8', 'spread': '1.0', 'groupId': 'OG000'}, {'value': '-13.1', 'spread': '1.0', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '32', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-11.2', 'spread': '1.0', 'groupId': 'OG000'}, {'value': '-13.3', 'spread': '1.0', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Papillomacular bundle thickness was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).', 'unitOfMeasure': 'µm', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in ETDRS Total Macular Volume', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '36', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.578', 'spread': '0.041', 'groupId': 'OG000'}, {'value': '-0.708', 'spread': '0.040', 'groupId': 'OG001'}]}]}, {'title': 'Week 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.686', 'spread': '0.048', 'groupId': 'OG000'}, {'value': '-0.782', 'spread': '0.048', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '33', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-0.720', 'spread': '0.050', 'groupId': 'OG000'}, {'value': '-0.800', 'spread': '0.050', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Early Treatment Diabetic Retinopathy Study (ETDRS) total macular volume was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).', 'unitOfMeasure': 'mm^3', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in the Foveal Threshold Sensitivities Obtained With HVF Analyzer II', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '3.5', 'spread': '12.3', 'groupId': 'OG000'}, {'value': '-7.4', 'spread': '12.8', 'groupId': 'OG001'}]}]}, {'title': 'Week 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '6.3', 'spread': '7.8', 'groupId': 'OG000'}, {'value': '-5.1', 'spread': '11.9', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '3.3', 'spread': '12.7', 'groupId': 'OG000'}, {'value': '1.4', 'spread': '19.5', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'The assessment of standardized automated visual fields was measured using the Humphrey Visual Field (HVF) Analyzer II. Automated visual fields included the assessment of foveal threshold sensitivities. Foveal threshold sensitivity is measured in decibels (dB), which ranges from 0 dB to 50 dB. A sensitivity threshold of 0 dB indicates not being able to see the most intense perimetric stimulus, while higher dB indicates better/normal foveal vision. A positive change from baseline indicates an improvement of symptoms.', 'unitOfMeasure': 'decibels (dB)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Visual Field Mean Deviation in Decibels of Sensitivity Obtained With HVF Analyzer II', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Baseline MD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-16.26', 'spread': '10.59', 'groupId': 'OG000'}, {'value': '-16.73', 'spread': '11.48', 'groupId': 'OG001'}]}]}, {'title': 'Week 48 MD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '37', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-24.26', 'spread': '9.37', 'groupId': 'OG000'}, {'value': '-23.76', 'spread': '10.40', 'groupId': 'OG001'}]}]}, {'title': 'Week 72 MD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-23.33', 'spread': '9.41', 'groupId': 'OG000'}, {'value': '-22.94', 'spread': '10.11', 'groupId': 'OG001'}]}]}, {'title': 'Week 96 MD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '33', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-23.31', 'spread': '9.41', 'groupId': 'OG000'}, {'value': '-22.70', 'spread': '9.88', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'The assessment of standardized automated visual fields was measured using the Humphrey Visual Field (HVF) Analyzer II. Automated visual fields included the assessment of the mean deviation (MD) in decibels (dB) of sensitivity.', 'unitOfMeasure': 'decibels (dB)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Contrast Sensitivity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '38', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48', 'categories': [{'measurements': [{'value': '-0.35', 'spread': '0.07', 'groupId': 'OG000'}, {'value': '-0.33', 'spread': '0.07', 'groupId': 'OG001'}]}]}, {'title': 'Week 72', 'categories': [{'measurements': [{'value': '-0.25', 'spread': '0.07', 'groupId': 'OG000'}, {'value': '-0.28', 'spread': '0.07', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'categories': [{'measurements': [{'value': '-0.27', 'spread': '0.07', 'groupId': 'OG000'}, {'value': '-0.25', 'spread': '0.07', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'The assessment of contrast sensitivity was measured using the Pelli-Robson chart. The chart uses letters arranged in groups whose contrast varies from high to low. Participants read the letters, starting with the highest contrast, until they are unable to read 2 or 3 letters in a single group. Each eye is assigned a score based on the contrast of the last group in which 2 or 3 letters were correctly read, ranging from 0 to 2.2 "log of contrast sensitivity" (LogCS) units. A score of 2.0 LogCS, represents a normal sensitivity contrast, and indicates the eye was able to detect 2 of the 3 letters with a contrast of 1 percent (contrast sensitivity = 100 percent or log 2). Scores less than 2.0 signify poorer contrast sensitivity. Pelli-Robson contrast sensitivity score of less than 1.5 is consistent with visual impairment and a score of less than 1.0 represents in visual disability. A negative change from baseline indicates worsening in symptoms.', 'unitOfMeasure': 'LogCS', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Color Vision', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '21', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'GS010-treated Eyes', 'description': 'All eyes that received the study treatment, GS010. Each participant was randomly assigned GS010 in either the right or left eye. The eye assigned to GS010 received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 μL. The injection was performed in the vitreous humor under local anesthesia.\n\nThe same participants also received the sham procedure in the right or left eye, which was not assigned to receive GS010, at the same study visit.'}, {'id': 'OG001', 'title': 'Sham-treated Eyes', 'description': 'All eyes that received the Sham. Each participant was randomly assigned GS010 in either the right or left eye, the eye not assigned GS010 received the sham procedure. The eye assigned to the Sham received one single sham intravitreal (IVT) injection, which was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.\n\nThe same participants also received GS010 in the right or left eye, which did not receive the sham procedure, at the same study visit.'}], 'classes': [{'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '133.1', 'spread': '290.7', 'groupId': 'OG000'}, {'value': '235.6', 'spread': '392.3', 'groupId': 'OG001'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}, {'units': 'Eyes', 'counts': [{'value': '21', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '97.7', 'spread': '342.2', 'groupId': 'OG000'}, {'value': '213.5', 'spread': '393.1', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 48 and Week 96', 'description': 'The assessment of color vision was measured using the Farnsworth-Munsell 100-Hue Color Test. Each of the 4 trays consisted of 21 caps. Participants were asked to sort the randomly arranged caps following the hue order from the first to the last fixed caps. The total error score (TES) was derived by the frequency the caps were misplaced and the severity, or distance of the misplacement.\n\nErrors were made whenever caps were misplaced from the correct order. Error scores were calculated according to the distance between any two caps. The error score for each individual cap was the sum of the difference between the number of that cap and the numbers of the cap adjacent to it, minus 2. TES was the total sum of the error scores of the entire set of caps.\n\nThe best possible score was 0 and there is no defined upper limit to the total error score range. A lower score indicates improved color discrimination ability. A positive change from baseline indicates a worsening in symptoms.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Eyes', 'denomUnitsSelected': 'Eyes', 'populationDescription': 'Intent-to-treat (ITT) population: All participants and all eyes that received study treatments, with data at both Baseline and the applicable post-dose visit. 1 participant was excluded from the ITT population due to receiving a smaller volume of study treatment than specified in the protocol. Participants received GS010 and Sham simultaneously.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'All Participants', 'description': 'All participants who were enrolled and received both study treatments, GS010 and Sham. All participants in the study received both GS010 and the sham procedure simultaneously. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham procedure in the eye not assigned to receive GS010, at the same study visit.\n\nGS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia.\n\nSham: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '39'}]}, {'type': 'Completed Week 48', 'achievements': [{'groupId': 'FG000', 'numSubjects': '38'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '35'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}], 'dropWithdraws': [{'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'All Participants', 'description': 'All participants who were enrolled and received both study treatments, GS010 and Sham. All participants in the study received both GS010 and the sham procedure simultaneously. Participants were randomly assigned to receive GS010 in either the right or left eye. The same participants also received the sham procedure in the eye not assigned to receive GS010, at the same study visit.\n\nGS010: Either the right or left eye received one single dose of GS010 (9E10 vg/eye) via an intravitreal (IVT) injection. The volume of the injected formula was 90 µL. The injection was performed in the vitreous humor under local anesthesia.\n\nSham: Either the right or left eye (the eye not randomly assigned to GS010) received the sham procedure. One single sham IVT injection was performed by applying pressure to the eye at the location of a typical IVT injection procedure, using the blunt end of a syringe without a needle.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '36.3', 'spread': '15.5', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '32', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}, {'title': 'Weight', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '73.9', 'spread': '17.8', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kilograms', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Height', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '174.5', 'spread': '7.7', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'centimeters', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-11-07', 'size': 1929780, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-01-17T11:48', 'hasProtocol': True}, {'date': '2018-12-21', 'size': 1612011, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_000.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-01-17T11:47', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 39}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-02-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'nctId': 'NCT03672968', 'statusForNctId': 'NO_LONGER_AVAILABLE', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2020-01', 'completionDateStruct': {'date': '2019-07-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-07-25', 'studyFirstSubmitDate': '2016-01-07', 'resultsFirstSubmitDate': '2020-01-17', 'studyFirstSubmitQcDate': '2016-01-08', 'lastUpdatePostDateStruct': {'date': '2022-07-29', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-01-17', 'studyFirstPostDateStruct': {'date': '2016-01-12', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-01-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-08-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48', 'timeFrame': 'Baseline and Week 48', 'description': 'Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Visual acuity is measured in "logarithm of the minimal angle of resolution" (LogMAR), which was derived from the number of letters participants could read on the ETDRS chart.\n\n1 ETDRS line = 5 letters\n\n1 ETDRS line = 0.1 LogMAR\n\nA lower LogMAR score denotes better visual acuity. A positive change from baseline indicates a worsening in symptoms.\n\nChange = (Week 48 score - Baseline score).'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in ETDRS Visual Acuity (Quantitative Score)', 'timeFrame': 'Baseline; Week 72 and Week 96', 'description': 'Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Visual acuity is measured in "logarithm of the minimal angle of resolution" (LogMAR), which was derived from the number of letters participants could read on the ETDRS chart.\n\n1 ETDRS line = 5 letters\n\n1 ETDRS line = 0.1 LogMAR\n\nA lower LogMAR score denotes better visual acuity. A positive change from baseline indicates a worsening in symptoms.\n\nChange = (Week 72 score - Baseline score) or (Week 96 score - Baseline score). Missing data was imputed by the linear interpolation method.'}, {'measure': 'Number of Eye Responders to Treatment', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'An eye was determined as a responder to treatment based on 2 different definitions.\n\nDefinition 1: An eye responder was defined by an improvement of the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity score of at least 15 letters compared to Baseline, or a final visual acuity greater than a Snellen acuity equivalent of 20/200 (a score of at least 1 letter).\n\nDefinition 2: An eye responder was defined by an improvement of the ETDRS score of at least 20 letters compared to Baseline.'}, {'measure': 'Number of Subject Responders to Treatment', 'timeFrame': 'Week 48; Week 72 and Week 96', 'description': 'A subject responder was defined as a participant whose Early Treatment Diabetic Retinopathy Study (ETDRS) score of the treated eye (that received GS010), was at least 15 letters better than the sham eye, or whose treated eye had a "logarithm of the minimal angle of resolution" (LogMAR) acuity score of at least 0.3 LogMAR better than the sham eye.\n\nFor the Week 96 analysis, if no score was available for Week 96, the score from the previous visit was used.'}, {'measure': 'Change From Baseline in GCL Macular Volume', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Ganglion cell layer (GCL) macular volume was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).'}, {'measure': 'Change From Baseline in RNFL Temporal Quadrant Thickness', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Retinal nerve fiber layer (RNFL) temporal quadrant thickness was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).'}, {'measure': 'Change From Baseline in RNFL Papillomacular Bundle Thickness', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Papillomacular bundle thickness was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).'}, {'measure': 'Change From Baseline in ETDRS Total Macular Volume', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'Early Treatment Diabetic Retinopathy Study (ETDRS) total macular volume was measured as a parameter of spectral domain-optical coherence tomography (SD-OCT). SD-OCT was obtained with the Spectralis® OCT (Heidelberg Engineering).'}, {'measure': 'Change From Baseline in the Foveal Threshold Sensitivities Obtained With HVF Analyzer II', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'The assessment of standardized automated visual fields was measured using the Humphrey Visual Field (HVF) Analyzer II. Automated visual fields included the assessment of foveal threshold sensitivities. Foveal threshold sensitivity is measured in decibels (dB), which ranges from 0 dB to 50 dB. A sensitivity threshold of 0 dB indicates not being able to see the most intense perimetric stimulus, while higher dB indicates better/normal foveal vision. A positive change from baseline indicates an improvement of symptoms.'}, {'measure': 'Visual Field Mean Deviation in Decibels of Sensitivity Obtained With HVF Analyzer II', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'The assessment of standardized automated visual fields was measured using the Humphrey Visual Field (HVF) Analyzer II. Automated visual fields included the assessment of the mean deviation (MD) in decibels (dB) of sensitivity.'}, {'measure': 'Change From Baseline in Contrast Sensitivity', 'timeFrame': 'Baseline; Week 48; Week 72 and Week 96', 'description': 'The assessment of contrast sensitivity was measured using the Pelli-Robson chart. The chart uses letters arranged in groups whose contrast varies from high to low. Participants read the letters, starting with the highest contrast, until they are unable to read 2 or 3 letters in a single group. Each eye is assigned a score based on the contrast of the last group in which 2 or 3 letters were correctly read, ranging from 0 to 2.2 "log of contrast sensitivity" (LogCS) units. A score of 2.0 LogCS, represents a normal sensitivity contrast, and indicates the eye was able to detect 2 of the 3 letters with a contrast of 1 percent (contrast sensitivity = 100 percent or log 2). Scores less than 2.0 signify poorer contrast sensitivity. Pelli-Robson contrast sensitivity score of less than 1.5 is consistent with visual impairment and a score of less than 1.0 represents in visual disability. A negative change from baseline indicates worsening in symptoms.'}, {'measure': 'Change From Baseline in Color Vision', 'timeFrame': 'Baseline; Week 48 and Week 96', 'description': 'The assessment of color vision was measured using the Farnsworth-Munsell 100-Hue Color Test. Each of the 4 trays consisted of 21 caps. Participants were asked to sort the randomly arranged caps following the hue order from the first to the last fixed caps. The total error score (TES) was derived by the frequency the caps were misplaced and the severity, or distance of the misplacement.\n\nErrors were made whenever caps were misplaced from the correct order. Error scores were calculated according to the distance between any two caps. The error score for each individual cap was the sum of the difference between the number of that cap and the numbers of the cap adjacent to it, minus 2. TES was the total sum of the error scores of the entire set of caps.\n\nThe best possible score was 0 and there is no defined upper limit to the total error score range. A lower score indicates improved color discrimination ability. A positive change from baseline indicates a worsening in symptoms.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Leber Hereditary Optic Neuropathy', 'Leber Hereditary Optic Atrophy', 'Heredity Optic Atrophy', 'Eye Diseases', 'Hereditary Eye Diseases', 'Inborn Genetic Disease', 'Genetic Therapy', 'Intravitreal Injections', 'Mitochondrial Disease', 'AAV2 Vectors', 'Nervous System Diseases', 'Neurodegenerative Disease', 'Heredodegenerative Disorders of the Nervous System'], 'conditions': ['Optic, Atrophy, Hereditary, Leber']}, 'referencesModule': {'references': [{'pmid': '36449262', 'type': 'DERIVED', 'citation': 'Carelli V, Newman NJ, Yu-Wai-Man P, Biousse V, Moster ML, Subramanian PS, Vignal-Clermont C, Wang AG, Donahue SP, Leroy BP, Sergott RC, Klopstock T, Sadun AA, Rebolleda Fernandez G, Chwalisz BK, Banik R, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA; the LHON Study Group. Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation. Ophthalmol Ther. 2023 Feb;12(1):401-429. doi: 10.1007/s40123-022-00611-x. Epub 2022 Nov 30.'}, {'pmid': '34108929', 'type': 'DERIVED', 'citation': 'Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.'}, {'pmid': '33451738', 'type': 'DERIVED', 'citation': 'Newman NJ, Yu-Wai-Man P, Carelli V, Moster ML, Biousse V, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Barboni P, DeBusk AA, Girmens JF, Rudolph G, Karanjia R, Taiel M, Blouin L, Smits G, Katz B, Sahel JA; LHON Study Group. Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset. Ophthalmology. 2021 May;128(5):649-660. doi: 10.1016/j.ophtha.2020.12.012. Epub 2021 Jan 12.'}], 'seeAlsoLinks': [{'url': 'http://ghr.nlm.nih.gov/condition/leber-hereditary-optic-neuropathy', 'label': "National Library of Medicine's Genetics Home Reference for Leber Hereditary Optic Neuropathy"}, {'url': 'https://www.nlm.nih.gov/medlineplus/genesandgenetherapy.html', 'label': "National Library of Medicine's Genes and Gene Therapy"}]}, 'descriptionModule': {'briefSummary': 'The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '15 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\nSelection Criteria:\n\nParticipants must meet all the following criteria at the Screening Visit (Visit 1) in order to be included into the study.\n\n1. Age 15 years or older.\n2. Onset of vision loss based on medically documented history or participant testimony, in at least one eye for ≤180 days in duration and if both eyes are affected the duration of vision loss in both eyes must be ≤180 days in duration.\n3. Each eye of the participant maintaining visual ability to allow at least for counting of the examiner's fingers at any distance.\n4. Female participants (if of childbearing potential) must agree to use effective methods of birth control up to 6 months after intravitreal (IVT) injection and male participants must agree to use condoms for up to 6 months after IVT injection.\n5. Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing.\n6. Signed written informed consent.\n\nInclusion Criteria:\n\nParticipants included in the study must satisfy all the following criteria at the Inclusion Visit (Visit 2).\n\n1. Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary Leber Hereditary Optical Neuropathy (LHON)-associated mutations (ND1 or ND6) in the participant's mitochondrial DNA.\n2. Review of all selection criteria to ensure continued compliance.\n3. Have a negative test for infection with human immunodeficiency virus.\n4. Have a negative pregnancy test for women of childbearing potential (a woman who is 2 years post-menopausal or surgically sterile is not considered to be of childbearing potential).\n\nExclusion Criteria:\n\nNon-Selection Criteria:\n\nParticipants who meet at least one of the following criteria at the Screening Visit (Visit 1) will not be included into the study.\n\n1. Any known allergy or hypersensitivity to GS010 or its constituents.\n2. Contraindication to IVT injection.\n3. IVT drug delivery to either eye within 30 days prior to the Screening Visit (Visit 1).\n4. Previous vitrectomy in either eye.\n5. Narrow angle in either eye contra-indicating pupillary dilation.\n6. Presence of disorders of the ocular media, such as the cornea and lens, which may interfere with visual acuity and other ocular assessments during the study period.\n7. Vision disorders, other than LHON, involving visual disability or with the potential to cause further vision loss during the trial period.\n8. Causes of optic neuropathy other than LHON and glaucoma.\n9. Participants with known mutations of other genes involved in pathological retinal or optic nerve conditions.\n10. Presence of ocular or systemic disease, other than LHON, whose pathology or associated treatments might affect the retina or the optic nerve.\n11. History of amblyopia associated with a Snellen visual acuity equivalent of worse than 20/80 (equivalent to 6/24 at 6 meters, decimal acuity 0.25, LogMAR +0.6) in the affected eye.\n12. Presence of ocular conditions, which in the opinion of the Investigator will prevent good quality SD-OCT imaging from being obtained.\n13. Presence, in either eye, of uncontrolled glaucoma, defined as an intra-ocular pressure (IOP) greater than 25 mmHg, despite maximal medical therapy with IOP-lowering agents.\n14. Active ocular inflammation or history of idiopathic or autoimmune-associated uveitis.\n15. Participants participating in another clinical trial and receiving an investigational medicinal product within 90 days prior to the Screening Visit (Visit 1).\n16. Previous treatment with an ocular gene therapy product.\n17. Participants who have undergone ocular surgery of clinical relevance (per Investigator opinion) within 90 days preceding the Screening Visit (Visit 1).\n18. Female participants who are or who intend to breast feed during the trial period.\n\nExclusion Criteria:\n\nParticipants who meet at least one of the following criteria at the Inclusion Visit (Visit 2) will not be included in the study.\n\n1. Any non-selection criteria which may have appeared after the Screening visit.\n2. Participants taking idebenone who have not completely discontinued the idebenone at least 7 days prior to Visit 2. If the participant has not discontinued idebenone at least 7 days prior to Visit 2, the visit may be delayed until the 7-day period is complete.\n3. Presence, at the time of study inclusion, of infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye.\n4. Presence of systemic illness, including alcohol and drug abuse (except nicotine), or medically significant abnormal laboratory values that are deemed by the Investigator to preclude the participant's safe participation in the study.\n5. Presence of illness or disease that, in the opinion of the Investigator, include symptoms and/or the associated treatments that can alter visual function, for instance cancers or pathology of the central nervous system.\n6. Any medical or psychological condition that, in the opinion of the Investigator, may compromise the safe participation of the participant in the study or would preclude compliance with the study protocol or ability of the participant to successfully complete the study.\n7. Participants unable or unwilling to comply with the protocol requirements."}, 'identificationModule': {'nctId': 'NCT02652767', 'acronym': 'RESCUE', 'briefTitle': 'Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'GenSight Biologics'}, 'officialTitle': 'A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene', 'orgStudyIdInfo': {'id': 'GS-LHON-CLIN-03A'}, 'secondaryIdInfos': [{'id': '2015-001265-11', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'GS010-treated Eyes', 'description': 'Each participant will have one eye randomly selected to receive a single injection of GS010 and the other eye will receive a sham injection. GS010-treated Eyes: GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). Participants will receive a single dose of GS010 in one of their randomly selected eyes, via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) plus 0.001% Pluronic F68®.', 'interventionNames': ['Biological: GS010']}, {'type': 'SHAM_COMPARATOR', 'label': 'Sham-treated Eyes', 'description': 'Each participant will have one eye randomly selected to receive GS010 and the other eye will receive a sham injection. Eyes receiving sham injection will undergo the same preparatory procedures as eyes receiving GS010 injection, including pupillary dilation, topical anti-infection and topical anesthetic procedures. Sham intravitreal injection will be performed by applying pressure to the eye at the location of a typical intravitreal injection procedure using the blunt end of a syringe without a needle.', 'interventionNames': ['Device: Sham Intravitreal Injection']}], 'interventions': [{'name': 'GS010', 'type': 'BIOLOGICAL', 'otherNames': ['Lenadogene Nolparvovec', 'rAAV2/2-ND4'], 'description': 'Both eyes of each participant will receive standard antiseptic preparation, administration of topical local ocular anesthetic agents and will undergo pupillary dilation. Administration of an intra-ocular pressure lowering agent will precede treatment for every participant.\n\nGS010-treated Eyes: GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). Participants will receive a single dose of GS010 in one of their randomly selected eyes, via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) plus 0.001% Pluronic F68®.', 'armGroupLabels': ['GS010-treated Eyes']}, {'name': 'Sham Intravitreal Injection', 'type': 'DEVICE', 'description': 'Both eyes of each participant will receive standard antiseptic preparation, administration of topical local ocular anesthetic agents and will undergo pupillary dilation. Administration of an intra-ocular pressure lowering agent will precede treatment for every participant.\n\nSham-treated Eyes: One eye of each participant will undergo sham injection. Sham intravitreal injection will be performed by applying pressure to the eye at the location of a typical intravitreal injection procedure using the blunt end of a syringe without a needle.', 'armGroupLabels': ['Sham-treated Eyes']}]}, 'contactsLocationsModule': {'locations': [{'zip': '91105', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Doheny Eye Center, University of California, Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Department of Ophthalmology, Emory University School of Medicine', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '19107', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'city': 'Paris', 'country': 'France', 'facility': "Centre National Hospitalier d'Ophtalmologie des Quinze-VingtCentre National Hospitalier d'Ophtalmologie des Quinze-Vingt", 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '80336', 'city': 'Munich', 'country': 'Germany', 'facility': 'Department of Neurology, University of Munich, Friedrich-Baur-Institute', 'geoPoint': {'lat': 48.13743, 'lon': 11.57549}}, {'city': 'Bologna', 'country': 'Italy', 'facility': 'IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria', 'geoPoint': {'lat': 44.49381, 'lon': 11.33875}}, {'zip': 'EC1V 2PD', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Moorfields Eye Hospital NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Nancy J. Newman, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Department of Ophthalmology, Emory University School of Medicine'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'GenSight Biologics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}