Ignite Creation Date:
2025-12-25 @ 2:13 AM
Ignite Modification Date:
2025-12-31 @ 10:10 PM
Study NCT ID:
NCT02695160
Status:
TERMINATED
Last Update Posted:
2024-07-19
First Post:
2016-02-24
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United Kingdom']}, 'conditionBrowseModule': {'meshes': [{'id': 'D002836', 'term': 'Hemophilia B'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@sangamo.com', 'phone': '+1 510 307 7266', 'title': 'Medical Monitor', 'organization': 'Sangamo Therapeutics'}, 'certainAgreement': {'otherDetails': 'All proposed written materials related to the study or an outline of proposed oral presentations, shall be submitted to Sangamo for approval at least 30 days prior to submission of materials for publication or oral disclosure to a third party. If Sangamo determines that a description of patentable subject matter is contained in written material or outline, it shall notify the clinical site within 1 month after receipt and Sangamo will have an additional 60 days for further review and action.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Due to the limited sample size of 1 subject, the primary and secondary endpoints could not be analyzed, and this study could not report any conclusions.'}}, 'adverseEventsModule': {'timeFrame': 'Up to 36 months after the SB-FIX infusion', 'eventGroups': [{'id': 'EG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.', 'otherNumAtRisk': 1, 'deathsNumAtRisk': 1, 'otherNumAffected': 1, 'seriousNumAtRisk': 1, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Sinus Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Chills', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment Related Adverse Events in Subjects Who Received SB-FIX as Assessed by Common Terminology Criteria for Adverse Events (CTCAE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'title': 'Number of participants with adverse events', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Number of participants with serious adverse events', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Up to 36 months after the SB-FIX infusion', 'description': 'Number of Participants with Treatment Related Adverse Events in Subjects Who Received SB-FIX as Assessed by Common Terminology Criteria for Adverse Events (CTCAE).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Factor 9 Antigen Levels Measured in IU/mL and Factor 9 Activity Levels Measured in IU/mL at Week 28 After SB-FIX Infusion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'title': 'Change from baseline of FIX activity', 'categories': [{'measurements': [{'value': '0.003', 'groupId': 'OG000'}]}]}, {'title': 'Change from baseline of FIX antigen levels', 'categories': [{'measurements': [{'value': '-0.006', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From screening through to week 28 after SB-FIX infusion', 'description': 'FIX antigen levels measured in IU/mL using Enzyme-Linked Immunosorbent Assay (ELISA).\n\nFIX activity levels measured in IU/mL using One-Stage Clot.', 'unitOfMeasure': 'IU/mL', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}, {'type': 'SECONDARY', 'title': 'Use of Factor IX Replacement Therapy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'title': 'Total factor replacements', 'categories': [{'measurements': [{'value': '170', 'groupId': 'OG000'}]}]}, {'title': 'Of which, purpose of treatment was recorded as prophylactic', 'categories': [{'measurements': [{'value': '45', 'groupId': 'OG000'}]}]}, {'title': 'Of which, purpose of treatment was not recorded', 'categories': [{'measurements': [{'value': '125', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From baseline through 36 months after the SB-FIX infusion', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.', 'unitOfMeasure': 'Infusion', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}, {'type': 'SECONDARY', 'title': 'Frequency and Severity of Bleeding Episodes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'title': 'Moderate bleeding episodes', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild bleeding episodes', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From baseline through 36 months after the SB-FIX infusion', 'description': 'The number and severity of bleeding events were collected from 3 weeks post-SB-FIX treatment, and for 120 weeks thereafter.', 'unitOfMeasure': 'Events', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}, {'type': 'SECONDARY', 'title': 'Immune Response to FIX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Change from baseline through 28 weeks after the SB-FIX infusion', 'description': 'Neutralizing antibodies to FIX measured by FIX inhibitor levels using Nijmegen-Bethesda assays.', 'unitOfMeasure': 'BU/mL', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}, {'type': 'SECONDARY', 'title': 'Presence of Shedding of AAV2/6 Vector DNA (in Copies/10 µL) by PCR in Plasma', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'title': 'Plasma: AAV2/6-ZFN42906 at Week 20: <10 copies/10 μL', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Plasma: AAV2/6-hF9 at Week 20: <10 copies/10 μL', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From baseline through week 20 after SB-FIX infusion', 'description': 'Two laboratory tests were run: AAV2/6-ZFN 42906 and AAV2/6-hF9. Presence in plasma was measured in number of copies/10 µL of whole plasma.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}, {'type': 'SECONDARY', 'title': 'Presence and Shedding of AAV2/6 Vector DNA (in Copies/100ng) by PCR in Saliva, Stool and Semen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'title': 'Saliva: AAV2/6-ZFN42906 at week12: <10 copies/100ng', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Saliva: AAV2/6-hF9 at week 12: <10 copies/100ng', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Stool: AAV2/6-ZFN42906 at week 12: <10 copies/100ng', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Stool: AAV2/6-hF9 at week 12: <10 copies/100ng', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Semen: AAV2/6-ZFN42906 at week 20: <20 copies/100ng', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Semen: AAV2/6-hF9 at week 20: <20 copies/100ng', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From baseline through week 20 after SB-FIX infusion', 'description': 'Subject data were collected at baseline and post infusion. Two laboratory tests were run for each sample type: AAV2/6-ZFN 42906 and AAV2/6-hF9. The presence of AAV2/6 vector DNA in saliva and stool was measured in number of copies/100 ng of sample DNA.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}, {'type': 'SECONDARY', 'title': 'Presence and Shedding of AAV2/6 Vector DNA (in Copies/250 µL) by PCR in Urine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'classes': [{'title': 'Urine: AAV2/6-ZFN42906 at Week 12: <10 copies/250 μL', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Urine: AAV2/6-hF9 at Week 12: <10 copies/250 μL', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From baseline through week 12 after SB-FIX infusion', 'description': 'Two laboratory tests were run: AAV2/6-ZFN 42906 and AAV2/6-hF9. Its presence in urine was measured in number of copies/250 µL of whole urine.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'High Dose', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '60', 'spread': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'calculatePct': False, 'unitOfMeasure': 'Participants'}, {'title': 'Height', 'classes': [{'categories': [{'measurements': [{'value': '173', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'cm'}, {'title': 'Weight', 'classes': [{'categories': [{'measurements': [{'value': '74.7', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'kg'}], 'populationDescription': 'All subjects in this study who received any portion of the SB-FIX infusion'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-03-20', 'size': 1754181, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-04-14T04:55', 'hasProtocol': True}, {'date': '2020-08-10', 'size': 335557, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-04-14T04:57', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 1}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2016-11-15', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-02', 'completionDateStruct': {'date': '2021-04-19', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-02-07', 'studyFirstSubmitDate': '2016-02-24', 'resultsFirstSubmitDate': '2022-04-14', 'studyFirstSubmitQcDate': '2016-02-24', 'lastUpdatePostDateStruct': {'date': '2024-07-19', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-02-07', 'studyFirstPostDateStruct': {'date': '2016-03-01', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2024-07-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-04-19', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment Related Adverse Events in Subjects Who Received SB-FIX as Assessed by Common Terminology Criteria for Adverse Events (CTCAE)', 'timeFrame': 'Up to 36 months after the SB-FIX infusion', 'description': 'Number of Participants with Treatment Related Adverse Events in Subjects Who Received SB-FIX as Assessed by Common Terminology Criteria for Adverse Events (CTCAE).'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Factor 9 Antigen Levels Measured in IU/mL and Factor 9 Activity Levels Measured in IU/mL at Week 28 After SB-FIX Infusion', 'timeFrame': 'From screening through to week 28 after SB-FIX infusion', 'description': 'FIX antigen levels measured in IU/mL using Enzyme-Linked Immunosorbent Assay (ELISA).\n\nFIX activity levels measured in IU/mL using One-Stage Clot.'}, {'measure': 'Use of Factor IX Replacement Therapy', 'timeFrame': 'From baseline through 36 months after the SB-FIX infusion', 'description': 'Participants received a single intravenous infusion of SB-FIX which is formed of 3 components (ZFN1, ZFN2, and cDNA donor) in 200mL of diluent adjusted to 0.25% human serum albumin on day 0 over a period of 2-8 hours.'}, {'measure': 'Frequency and Severity of Bleeding Episodes', 'timeFrame': 'From baseline through 36 months after the SB-FIX infusion', 'description': 'The number and severity of bleeding events were collected from 3 weeks post-SB-FIX treatment, and for 120 weeks thereafter.'}, {'measure': 'Immune Response to FIX', 'timeFrame': 'Change from baseline through 28 weeks after the SB-FIX infusion', 'description': 'Neutralizing antibodies to FIX measured by FIX inhibitor levels using Nijmegen-Bethesda assays.'}, {'measure': 'Presence of Shedding of AAV2/6 Vector DNA (in Copies/10 µL) by PCR in Plasma', 'timeFrame': 'From baseline through week 20 after SB-FIX infusion', 'description': 'Two laboratory tests were run: AAV2/6-ZFN 42906 and AAV2/6-hF9. Presence in plasma was measured in number of copies/10 µL of whole plasma.'}, {'measure': 'Presence and Shedding of AAV2/6 Vector DNA (in Copies/100ng) by PCR in Saliva, Stool and Semen', 'timeFrame': 'From baseline through week 20 after SB-FIX infusion', 'description': 'Subject data were collected at baseline and post infusion. Two laboratory tests were run for each sample type: AAV2/6-ZFN 42906 and AAV2/6-hF9. The presence of AAV2/6 vector DNA in saliva and stool was measured in number of copies/100 ng of sample DNA.'}, {'measure': 'Presence and Shedding of AAV2/6 Vector DNA (in Copies/250 µL) by PCR in Urine', 'timeFrame': 'From baseline through week 12 after SB-FIX infusion', 'description': 'Two laboratory tests were run: AAV2/6-ZFN 42906 and AAV2/6-hF9. Its presence in urine was measured in number of copies/250 µL of whole urine.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hemophilia B']}, 'referencesModule': {'references': [{'pmid': '36299240', 'type': 'DERIVED', 'citation': 'Harmatz P, Prada CE, Burton BK, Lau H, Kessler CM, Cao L, Falaleeva M, Villegas AG, Zeitler J, Meyer K, Miller W, Wong Po Foo C, Vaidya S, Swenson W, Shiue LH, Rouy D, Muenzer J. First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B. Mol Ther. 2022 Dec 7;30(12):3587-3600. doi: 10.1016/j.ymthe.2022.10.010. Epub 2022 Oct 25.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX. SB-FIX is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the Factor 9 gene into the albumin locus in hepatocytes with the goal of lifelong therapeutic production of the Factor IX clotting factor.', 'detailedDescription': "The objective of the study is to provide long term expression of Factor IX in subjects with severe hemophilia B. SB-FIX is a therapeutic for ZFN-mediated genome editing which will be delivered by adeno-associated virus (AAV)-derived vectors. SB-FIX is intended to function by placement of a corrective copy of the Factor IX transgene into the genome of the subject's own hepatocytes, under the control of the highly expressed endogenous albumin locus, and is expected to provide permanent, liver-specific expression of Factor IX for the lifetime of a hemophilia B subject."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male \\>18 years of age\n* Severe hemophilia B (native circulating FIX activity \\<1%, with or without cross reactive material)\n\nExclusion Criteria:\n\n* Presence of neutralizing antibodies\n* History of hypersensitivity response or an allergic reaction to FIX or FIX products\n* Currently receiving long acting FIX replacement therapy\n* FIX mutations known to be associated with FIX inhibitors\n* Polymorphisms in the ZFN target region\n* Presence of any liver mass on MRI, or elevated alpha-fetoprotein (AFP)\n* Any contraindication to the use of corticosteroids for immunosuppression\n* Currently receiving antiviral therapy for hepatitis B or C or with history or active hepatitis B or hepatitis C or HIV-1 or HIV1/2 antibody positive.\n* Chronic anemia, leukopenia, or thrombocytopenia\n* Past medical history of active tuberculosis or significant fungal disease\n* Symptomatic cardiovascular disease as a co-morbid condition\n* Markers of hepatic inflammation or overt or occult cirrhosis\n* History of chronic renal disease or creatinine ≥ 1.5 mg/dL\n* Systemic (iv or oral) immunomodulatory agent or steroid use (topical treatment is allowed)\n* History of chronic infection or other chronic disorder considered an unacceptable risk\n* History of malignancy except for treated basal cell or squamous cell carcinoma\n* History of alcohol or substance abuse\n* Previously received gene therapy product\n* Participation in prior investigational drug or medical device study within the previous 3 months\n* History of therapeutic non-adherence\n* Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study'}, 'identificationModule': {'nctId': 'NCT02695160', 'briefTitle': 'Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sangamo Therapeutics'}, 'officialTitle': 'A Phase I, Open-Label, Ascending Dose Study to Assess the Safety and Tolerability of AAV2/6 Factor IX Gene Therapy Via Zinc Finger Nuclease (ZFN) Mediated Targeted Integration of SB-FIX in Adult Subjects With Severe Hemophilia B', 'orgStudyIdInfo': {'id': 'SB-FIX-1501'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': 'SB-FIX: Low Dose', 'interventionNames': ['Biological: SB-FIX']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'SB-FIX: Medium Dose', 'interventionNames': ['Biological: SB-FIX']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'description': 'SB-FIX: High Dose', 'interventionNames': ['Biological: SB-FIX']}], 'interventions': [{'name': 'SB-FIX', 'type': 'BIOLOGICAL', 'description': 'Single dose of each of the 3 components of SB-FIX: ZFN1, ZFN2 and cDNA Donor.', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 3']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20007', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': 'Georgetown University Medical Center', 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}], 'overallOfficials': [{'name': 'Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Sangamo Therapeutics, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sangamo Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}