Ignite Creation Date:
2025-12-26 @ 11:13 PM
Ignite Modification Date:
2025-12-26 @ 11:13 PM
Study NCT ID:
NCT06271512
Status:
RECRUITING
Last Update Posted:
2025-02-11
First Post:
2024-02-14
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Bone marrow and whole blood collected at the time of bone marrow sample collection will be stored centrally to facilitate subsequent exploratory analyses that may include gene/genomic expression studies.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 150}, 'targetDuration': '15 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-01-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2043-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-02-10', 'studyFirstSubmitDate': '2024-02-14', 'studyFirstSubmitQcDate': '2024-02-14', 'lastUpdatePostDateStruct': {'date': '2025-02-11', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-02-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2043-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)', 'timeFrame': 'Through 15 years post-beti-cel infusion', 'description': 'The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event \\[SAE\\]):\n\n* Any newly diagnosed malignancy\n* Neutrophil engraftment failure: defined as healthcare provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure\n* Newly acquired human immunodeficiency virus-1 (HIV-1), HIV-2 infection and human t-lymphotropic virus (HTLV) infection\n* Any newly diagnosed autoimmune disorder\n* Any hepatic veno-occlusive disease (VOD)\n* Any clinically significant bleeding events.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants with Serious Adverse Events (SAEs)', 'timeFrame': 'Through 15 years post-beti-cel infusion'}, {'measure': 'Number of Participants with beti-cel related AEs', 'timeFrame': 'Through 15 years post-beti-cel infusion'}, {'measure': 'Event-Free Survival', 'timeFrame': 'Through 15 years post-beti-cel infusion'}, {'measure': 'Percentage of Participants Achieving Transfusion Independence', 'timeFrame': 'Through 15 years post-beti-cel infusion'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Beta-Thalassemia']}, 'descriptionModule': {'briefSummary': 'The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Participants with β-thalassemia treated with beti-cel in the post marketing setting at a center in the United States (US) that participates in the Registry.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry.\n* Participant must sign an informed consent and/or assent prior to enrollment as required under applicable laws and regulations.\n* Participant must have signed an informed consent and/or assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR).\n* Participant must be followed by a hematologist based in the US.\n\nExclusion Criteria:\n\n\\- There are no exclusion criteria for Registry participation.'}, 'identificationModule': {'nctId': 'NCT06271512', 'briefTitle': 'A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel', 'organization': {'class': 'INDUSTRY', 'fullName': 'Genetix Biotherapeutics Inc.'}, 'officialTitle': 'A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)', 'orgStudyIdInfo': {'id': 'REG-501'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'All Participants', 'description': 'Participants with β-thalassemia treated with beti-cel in the post-marketing setting will be followed in this registry study for up to 15 years after infusion with beti-cel to collect real-world longitudinal data.', 'interventionNames': ['Other: No Intervention']}], 'interventions': [{'name': 'No Intervention', 'type': 'OTHER', 'description': 'This is non-interventional study.', 'armGroupLabels': ['All Participants']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': "UCSF Benioff Children's Hospitals", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '94043', 'city': 'Palo Alto', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '11040', 'city': 'New Hyde Park', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Cohen Children's Medical Center", 'geoPoint': {'lat': 40.7351, 'lon': -73.68791}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'centralContacts': [{'name': 'bluebird bio', 'role': 'CONTACT', 'email': 'clinicaltrials@bluebirdbio.com', 'phone': '+1-833-999-6378'}], 'overallOfficials': [{'name': 'Himal Lal Thakar, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'bluebird bio, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': 'bluebird bio is committed to transparency. Appropriately de-identified participant-level datasets and supporting documents may be shared (if bluebird bio is contractually permitted to do so) following completion of this study, completion of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genetix Biotherapeutics Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Center for International Blood and Marrow Transplant Research', 'class': 'NETWORK'}], 'responsibleParty': {'type': 'SPONSOR'}}}}