Ignite Creation Date:
2025-12-24 @ 11:38 PM
Ignite Modification Date:
2026-01-07 @ 12:39 PM
Study NCT ID:
NCT01526356
Status:
COMPLETED
Last Update Posted:
2020-10-26
First Post:
2012-02-01
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Topical Rapamycin to Erase Angiofibromas in TSC
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D018322', 'term': 'Angiofibroma'}, {'id': 'D014402', 'term': 'Tuberous Sclerosis'}], 'ancestors': [{'id': 'D009383', 'term': 'Neoplasms, Vascular Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006222', 'term': 'Hamartoma'}, {'id': 'D009378', 'term': 'Neoplasms, Multiple Primary'}, {'id': 'D009386', 'term': 'Neoplastic Syndromes, Hereditary'}, {'id': 'D065703', 'term': 'Malformations of Cortical Development, Group I'}, {'id': 'D054220', 'term': 'Malformations of Cortical Development'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020752', 'term': 'Neurocutaneous Syndromes'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D020123', 'term': 'Sirolimus'}], 'ancestors': [{'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'mary.k.koenig@uth.tmc.edu', 'phone': '713-500-7142', 'title': 'Mary Kay Koenig, MD', 'organization': 'The University of Texas Health Science Center at Houston'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '6 months', 'description': 'Serious adverse events were determined to be unrelated to study drug.', 'eventGroups': [{'id': 'EG000', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.', 'otherNumAtRisk': 59, 'deathsNumAtRisk': 59, 'otherNumAffected': 10, 'seriousNumAtRisk': 59, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose', 'otherNumAtRisk': 63, 'deathsNumAtRisk': 63, 'otherNumAffected': 18, 'seriousNumAtRisk': 63, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG002', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose', 'otherNumAtRisk': 57, 'deathsNumAtRisk': 57, 'otherNumAffected': 22, 'seriousNumAtRisk': 57, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Application site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Application site pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Application site acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Cutaneous eruption', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Application site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Sunburn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Application site irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Application site papules', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Application site paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Mouth ulceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}], 'seriousEvents': [{'term': 'cholecystectomy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Subependymal Giant Cell Astrocytoma (SEGA) resection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 59, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 63, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 57, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}], 'frequencyThreshold': '1.1'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '47', 'groupId': 'OG000'}, {'value': '46', 'groupId': 'OG001'}, {'value': '40', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'OG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}], 'classes': [{'categories': [{'measurements': [{'value': '-13.89', 'spread': '20.86', 'groupId': 'OG000'}, {'value': '-12.71', 'spread': '20.24', 'groupId': 'OG001'}, {'value': '-1.39', 'spread': '14.64', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline, 6 months', 'description': "Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'This data was not collected for 12 in the 1% rapamycin arm, 17 in the 0.1% rapamycin arm, and 17 in the placebo arm.'}, {'type': 'SECONDARY', 'title': "Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient", 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}, {'value': '58', 'groupId': 'OG001'}, {'value': '51', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'OG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}], 'classes': [{'categories': [{'title': 'better', 'measurements': [{'value': '45', 'groupId': 'OG000'}, {'value': '38', 'groupId': 'OG001'}, {'value': '13', 'groupId': 'OG002'}]}, {'title': 'same', 'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}, {'value': '15', 'groupId': 'OG002'}]}, {'title': 'worse', 'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'baseline, 6 months', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'This data was not collected for 4 in the 1% rapamycin arm, 5 in the 0.1% rapamycin arm, and 6 in the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}, {'value': '29', 'groupId': 'OG001'}, {'value': '22', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'OG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.5', 'spread': '4.4', 'groupId': 'OG000'}, {'value': '-3.2', 'spread': '5.0', 'groupId': 'OG001'}, {'value': '-1.6', 'spread': '4.5', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline, 6 months', 'description': 'The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'This data was not collected for 29 in the 1% rapamycin arm, 34 in the 0.1% rapamycin arm, and 35 in the placebo arm.'}, {'type': 'SECONDARY', 'title': "Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)", 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '9', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'OG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.2', 'spread': '4.1', 'groupId': 'OG000'}, {'value': '-1.7', 'spread': '3.1', 'groupId': 'OG001'}, {'value': '-1.8', 'spread': '2.6', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline, 6 months', 'description': "The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'This data was not collected for 46 in the 1% rapamycin arm, 57 in the 0.1% rapamycin arm, and 48 in the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '28', 'groupId': 'OG001'}, {'value': '19', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'OG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.2', 'spread': '3.2', 'groupId': 'OG000'}, {'value': '-1.8', 'spread': '5.2', 'groupId': 'OG001'}, {'value': '0.3', 'spread': '3.4', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline, 6 months', 'description': 'The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'This data was not collected for 41 in the 1% rapamycin arm, 35 in the 0.1% rapamycin arm, and 38 in the placebo arm.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Number of Events of Dermatologic Sensitivity at the Site of Application', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '63', 'groupId': 'OG001'}, {'value': '57', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}, {'id': 'OG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'OG002', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}], 'classes': [{'title': 'Application site pain', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Application site pruritis', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Application site erythema', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '6 months', 'description': 'Dermatologic sensitivity includes pain, pruritis, or erythema at the application site.', 'unitOfMeasure': 'number of events', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Number of Participants With Systemic Uptake of Topically Applied Rapamycin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}, {'value': '63', 'groupId': 'OG001'}, {'value': '57', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'OG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 months', 'description': 'Blood levels checked to confirm the lack of systemic rapamycin.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'FG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'FG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '59'}, {'groupId': 'FG001', 'numSubjects': '63'}, {'groupId': 'FG002', 'numSubjects': '57'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '51'}, {'groupId': 'FG001', 'numSubjects': '53'}, {'groupId': 'FG002', 'numSubjects': '45'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '10'}, {'groupId': 'FG002', 'numSubjects': '12'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'BG000'}, {'value': '63', 'groupId': 'BG001'}, {'value': '57', 'groupId': 'BG002'}, {'value': '179', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': '1% Rapamycin', 'description': '1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. High Dose'}, {'id': 'BG001', 'title': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream\n\nRapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose'}, {'id': 'BG002', 'title': 'Placebo', 'description': 'Cream only\n\nPlacebo: Study cream is applied nightly to the affected areas on the face.'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '19.2', 'spread': '13.0', 'groupId': 'BG000'}, {'value': '21.7', 'spread': '12.4', 'groupId': 'BG001'}, {'value': '20.3', 'spread': '14.3', 'groupId': 'BG002'}, {'value': '20.5', 'spread': '13.2', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '36', 'groupId': 'BG000'}, {'value': '34', 'groupId': 'BG001'}, {'value': '29', 'groupId': 'BG002'}, {'value': '99', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '28', 'groupId': 'BG002'}, {'value': '80', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 179}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-09', 'completionDateStruct': {'date': '2014-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-09-30', 'studyFirstSubmitDate': '2012-02-01', 'resultsFirstSubmitDate': '2020-09-30', 'studyFirstSubmitQcDate': '2012-02-02', 'lastUpdatePostDateStruct': {'date': '2020-10-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-09-30', 'studyFirstPostDateStruct': {'date': '2012-02-03', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-10-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2014-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Number of Events of Dermatologic Sensitivity at the Site of Application', 'timeFrame': '6 months', 'description': 'Dermatologic sensitivity includes pain, pruritis, or erythema at the application site.'}, {'measure': 'Number of Participants With Systemic Uptake of Topically Applied Rapamycin', 'timeFrame': '6 months', 'description': 'Blood levels checked to confirm the lack of systemic rapamycin.'}], 'primaryOutcomes': [{'measure': 'Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score', 'timeFrame': 'baseline, 6 months', 'description': "Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome."}], 'secondaryOutcomes': [{'measure': "Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient", 'timeFrame': 'baseline, 6 months'}, {'measure': 'Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)', 'timeFrame': 'baseline, 6 months', 'description': 'The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).'}, {'measure': "Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)", 'timeFrame': 'baseline, 6 months', 'description': "The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life)."}, {'measure': 'Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)', 'timeFrame': 'baseline, 6 months', 'description': 'The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Angiofibroma', 'Tuberous Sclerosis', 'Rapamycin', 'Sirolimus', 'mTOR'], 'conditions': ['Angiofibromas', 'Tuberous Sclerosis']}, 'referencesModule': {'references': [{'pmid': '29800048', 'type': 'DERIVED', 'citation': 'Koenig MK, Bell CS, Hebert AA, Roberson J, Samuels JA, Slopis JM, Tate P, Northrup H; TREATMENT Trial Collaborators. Efficacy and Safety of Topical Rapamycin in Patients With Facial Angiofibromas Secondary to Tuberous Sclerosis Complex: The TREATMENT Randomized Clinical Trial. JAMA Dermatol. 2018 Jul 1;154(7):773-780. doi: 10.1001/jamadermatol.2018.0464.'}]}, 'descriptionModule': {'briefSummary': 'The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months.\n\nThe primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects must be willing and able to comply with all trial requirements.\n* Subject has a diagnosis of TSC and has visible facial angiofibromas.\n* Female subjects of child bearing potential must not be pregnant and must agree to use appropriate contraceptive methods .\n\nExclusion Criteria:\n\n* Subject is currently receiving therapy with Rapamycin.\n* Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction.\n* Subject is currently participating in or has participated within the last 30 days in a clinical trial involving an investigational drug.\n* Subject has a known hypersensitivity to either the vehicle or Rapamycin.\n* Subject is a pregnant or nursing female.\n* Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas.\n* Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.'}, 'identificationModule': {'nctId': 'NCT01526356', 'acronym': 'Treatment', 'briefTitle': 'Topical Rapamycin to Erase Angiofibromas in TSC', 'organization': {'class': 'OTHER', 'fullName': 'The University of Texas Health Science Center, Houston'}, 'officialTitle': 'Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy', 'orgStudyIdInfo': {'id': 'HSC-MS-11-0501'}, 'secondaryIdInfos': [{'id': 'Department of Defense USAMRMC', 'type': 'OTHER_GRANT', 'domain': 'W81XWH-11-1-0240'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Cream only', 'interventionNames': ['Drug: Placebo']}, {'type': 'ACTIVE_COMPARATOR', 'label': '0.1 % Rapamycin', 'description': '0.1% Rapamycin cream', 'interventionNames': ['Drug: Rapamycin']}, {'type': 'ACTIVE_COMPARATOR', 'label': '1% Rapamycin', 'description': '1% Rapamycin cream', 'interventionNames': ['Drug: Rapamycin']}], 'interventions': [{'name': 'Placebo', 'type': 'DRUG', 'otherNames': ['Rapamycin'], 'description': 'Study cream is applied nightly to the affected areas on the face.', 'armGroupLabels': ['Placebo']}, {'name': 'Rapamycin', 'type': 'DRUG', 'description': 'Study cream is applied nightly to the affected areas on the face. Low Dose', 'armGroupLabels': ['0.1 % Rapamycin']}, {'name': 'Rapamycin', 'type': 'DRUG', 'description': 'Study cream is applied nightly to the affected areas on the face. High Dose', 'armGroupLabels': ['1% Rapamycin']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35294', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "UCLA Mattel Children's Hospital", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Herscot Center for Adults and Children with TSC Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55102-2697', 'city': 'Saint Paul', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Clinic Without Walls', 'geoPoint': {'lat': 44.94441, 'lon': -93.09327}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '75219', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Texas Scottish Rite Hospital', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas Medical School at Houston', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'city': 'Sydney', 'state': 'New South Wales', 'country': 'Australia', 'facility': "Sydney Children's Hospital", 'geoPoint': {'lat': -33.86785, 'lon': 151.20732}}], 'overallOfficials': [{'name': 'Mary Kay Koenig, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'The University of Texas Medical School at Houston'}, {'name': 'Hope Northrup, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'The University of Texas Medical School at Houston'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'The University of Texas Health Science Center, Houston', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Associate Professor', 'investigatorFullName': 'Mary Kay Koenig', 'investigatorAffiliation': 'The University of Texas Health Science Center, Houston'}}}}