Ignite Creation Date:
2025-12-26 @ 2:40 PM
Ignite Modification Date:
2026-01-01 @ 5:52 AM
Study NCT ID:
NCT04925206
Status:
TERMINATED
Last Update Posted:
2025-03-30
First Post:
2021-06-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
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': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}}, 'statusModule': {'whyStopped': 'Development plan change', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2021-08-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2025-03-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-03-25', 'studyFirstSubmitDate': '2021-06-04', 'studyFirstSubmitQcDate': '2021-06-11', 'lastUpdatePostDateStruct': {'date': '2025-03-30', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-06-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-01-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Frequency and severity of collected AEs & SAEs', 'timeFrame': 'Within 24 months after ET-01 infusion'}, {'measure': 'All-cause mortality', 'timeFrame': 'From signing of informed consent up to 24 months post-ET-01 infusion'}, {'measure': 'Incidence of transplant-related mortality', 'timeFrame': 'From baseline (pre-transfusion) up to 12 months post-ET-01 infusion'}, {'measure': 'Total lymphocyte count', 'timeFrame': 'Within 24 months after ET-01 infusion'}, {'measure': 'Proportion of subjects with abnormal proliferation of blood cells', 'timeFrame': 'Within 24 months after ET-01 infusion'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Beta-Thalassaemia', 'CRISPR-Cas9', 'Human Hematopoietic Stem and Progenitor Cells'], 'conditions': ['Transfusion Dependent Beta-Thalassaemia']}, 'descriptionModule': {'briefSummary': 'This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '35 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Subjects and/or legal representative fully understand and voluntarily sign informed consent forms.\n* Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol.\n* Be not appropriate to receive conventional allogeneic hematopoietic stem cell transplantation.\n* Lansky/Karnofsky score ≥ 70%.\n* Eligible for hematopoietic stem cell transplantation and conditioning with busulfan as per investigator's judgement.\n\nOther protocol defined inclusion criteria may apply.\n\nKey Exclusion Criteria:\n\n* Subjects with associated α-thalassemia.\n* Subjects with any clinically significant acute or uncontrolled infections.\n* History of uncontrolled epilepsy or other mental disorders.\n* Previous treatment with allogeneic bone marrow transplantation or gene therapy.\n\nOther protocol defined exclusion criteria may apply."}, 'identificationModule': {'nctId': 'NCT04925206', 'acronym': 'ET-01', 'briefTitle': 'A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia', 'organization': {'class': 'INDUSTRY', 'fullName': 'EdiGene (GuangZhou) Inc.'}, 'officialTitle': 'A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia', 'orgStudyIdInfo': {'id': 'EDG-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ET-01', 'interventionNames': ['Biological: ET-01']}], 'interventions': [{'name': 'ET-01', 'type': 'BIOLOGICAL', 'description': 'Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.', 'armGroupLabels': ['ET-01']}]}, 'contactsLocationsModule': {'locations': [{'zip': '510515', 'city': 'Guangzhou', 'state': 'Guangdong', 'country': 'China', 'facility': 'Nanfang Hospital of Southern Medical University', 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}, {'zip': '510623', 'city': 'Guangzhou', 'state': 'Guangdong', 'country': 'China', 'facility': "Guangzhou Women and Children's Medical Center", 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}, {'zip': '518035', 'city': 'Shenzhen', 'state': 'Guangdong', 'country': 'China', 'facility': "Shenzhen Children's Hospital", 'geoPoint': {'lat': 22.54554, 'lon': 114.0683}}, {'zip': '300020', 'city': 'Tianjin', 'state': 'Tianjin Municipality', 'country': 'China', 'facility': 'Institute of Hematology & Blood Diseases Hospital', 'geoPoint': {'lat': 39.14222, 'lon': 117.17667}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'EdiGene (GuangZhou) Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}