Ignite Creation Date:
2025-12-24 @ 8:03 PM
Ignite Modification Date:
2026-01-04 @ 4:14 PM
Study NCT ID:
NCT02204904
Status:
TERMINATED
Last Update Posted:
2020-05-21
First Post:
2014-07-28
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2022-05-23', 'releaseDate': '2022-03-04'}], 'estimatedResultsFirstSubmitDate': '2022-03-04'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D000326', 'term': 'Adrenoleukodystrophy'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D018901', 'term': 'Peroxisomal Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000309', 'term': 'Adrenal Insufficiency'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 59}, 'patientRegistry': False}, 'statusModule': {'whyStopped': 'Sufficient enrollment for Month 24 safety and efficacy endpoints', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2015-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-12', 'completionDateStruct': {'date': '2019-12-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-05-19', 'studyFirstSubmitDate': '2014-07-28', 'studyFirstSubmitQcDate': '2014-07-29', 'lastUpdatePostDateStruct': {'date': '2020-05-21', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-07-31', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2019-12-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of transplant-related mortality (TRM).', 'timeFrame': 'Through 100 and 365 days post allo-HSC infusion', 'description': 'TRM is defined as death due to any transplantation-related cause other than disease progression.'}, {'measure': 'Incidence and timing of neutrophil engraftment.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Incidence and timing of platelet engraftment', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Incidence of engraftment failure or allograft rejection.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Incidence of primary donor-derived chimerism of ≥50%.', 'timeFrame': 'by 100 days post allo-HSC infusion'}, {'measure': 'Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Proportion of subjects who experience either ≥Grade II acute (Graft versus Host Disease) GVHD or chronic GVHD.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Incidence of ≥Grade II acute GVHD.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Incidence of chronic GVHD.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Number of emergency room visits.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Number and duration of intensive care unit stay.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}, {'measure': 'Number and duration of in-patient hospitalization.', 'timeFrame': '1-48 (± 1) months post allo-HSC infusion'}], 'secondaryOutcomes': [{'measure': 'Incidence of Major Functional Disabilities (MFDs).', 'timeFrame': '1-48 (± 2) months post allo-HSC infusion', 'description': 'MFDs is defined as any of the following: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement.'}, {'measure': 'Change from Baseline in Loes score', 'timeFrame': '1-48 (± 2) months post allo-HSC infusion'}, {'measure': 'Change from Baseline in Neurological Function Score (NFS)', 'timeFrame': '1-48 (± 2) months post allo-HSC infusion'}, {'measure': 'Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable', 'timeFrame': '1-48 (± 2) months post allo-HSC infusion'}, {'measure': 'MFD-free survival', 'timeFrame': '48 (± 2) months post allo-HSC infusion'}, {'measure': 'Overall survival', 'timeFrame': '48 (± 2) months post allo-HSC infusion'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['X-linked Adrenoleukodystrophy', 'Hematopoietic Stem Cells'], 'conditions': ['X-Linked Adrenoleukodystrophy (X-ALD)', 'Cerebral Adrenoleukodystrophy (CALD)', 'Adrenoleukodystrophy (ALD)']}, 'referencesModule': {'references': [{'pmid': '34781360', 'type': 'DERIVED', 'citation': 'Chiesa R, Boelens JJ, Duncan CN, Kuhl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy. Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294.'}]}, 'descriptionModule': {'briefSummary': 'Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Boys aged 17 or younger receiving allogeneic hematopoietic stem cell transplantation for the treatment of cerebral adrenoleukodystrophy prospectively or partially prospective/retrospective', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Provide informed consent from a competent custodial parent or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. In addition, informed assent will be sought from capable subjects, in accordance with the directive of the institution's IRB/IEC and all other local requirements.\n2. Be male and ≤17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at the time of parental/guardian consent and, where appropriate, subject assent, for prospective subjects.\n3. Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.\n4. Depending on the cohort, the subject must:\n\n * Be scheduled for allo-HSCT evaluation at a study site (prospective cohort only),\n * Have received an allo-HSC infusion and be consented in time to complete the Month 24 Visit on study (partial prospective/retrospective cohort only), or\n * Have received their most recent allo-HSC infusion on or after January 1, 2013 (retrospective cohort only).\n\nExclusion Criteria:\n\n1. Previous treatment with a gene therapy product.\n2. Receipt of an experimental transplantation procedure."}, 'identificationModule': {'nctId': 'NCT02204904', 'briefTitle': 'Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Genetix Biotherapeutics Inc.'}, 'officialTitle': 'A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in Males ≤17 Years of Age Undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy', 'orgStudyIdInfo': {'id': 'ALD-103'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Allo-HSCT prospective', 'description': 'Subjects who will be consented before they received an allo-HSC infusion. They will be consented and enrolled on the study during the Screening Period.', 'interventionNames': ['Genetic: Allo-HSCT']}, {'label': 'Allo-HSCT partial prospective/retrospective', 'description': 'Subjects who will be consented after they received an allo-HSC infusion but before they reach 24 months post-infusion on study. Subjects in this cohort will participate prospectively in at least the Month 24 Visit in order to obtain prospective on-study data for this and all visits after Month 24', 'interventionNames': ['Genetic: Allo-HSCT']}, {'label': 'Allo-HSCT retrospective', 'description': 'Subjects who received an allo-HSC infusion on or after January 1, 2013 and died before study data collection.', 'interventionNames': ['Genetic: Allo-HSCT']}], 'interventions': [{'name': 'Allo-HSCT', 'type': 'GENETIC', 'description': 'Allogeneic Hematopoietic Stem Cell Transplantation', 'armGroupLabels': ['Allo-HSCT partial prospective/retrospective', 'Allo-HSCT prospective', 'Allo-HSCT retrospective']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '02141', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital/Massachusetts General Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "The Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hospital Austral', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'zip': 'H3H 2R9', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'McGill University Health Centre', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'city': 'Leipzig', 'country': 'Germany', 'facility': 'University Hospital Leipzig', 'geoPoint': {'lat': 51.33962, 'lon': 12.37129}}, {'zip': '00165', 'city': 'Roma', 'country': 'Italy', 'facility': 'IRCCS Ospedale Pediatrico Bambine Gesú', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'city': 'Utrecht', 'country': 'Netherlands', 'facility': 'Princess Maxima Center for Pediatric Oncology (PMC)', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Central Manchester University Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'overallOfficials': [{'name': 'Elizabeth McNeil, MD MSc', 'role': 'STUDY_DIRECTOR', 'affiliation': 'bluebird bio, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genetix Biotherapeutics Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}, 'annotationSection': {'annotationModule': {'unpostedAnnotation': {'unpostedEvents': [{'date': '2022-03-04', 'type': 'RELEASE'}, {'date': '2022-05-23', 'type': 'RESET'}], 'unpostedResponsibleParty': 'Genetix Biotherapeutics Inc.'}}}}