Ignite Creation Date:
2025-12-25 @ 1:46 AM
Ignite Modification Date:
2025-12-31 @ 2:34 PM
Study NCT ID:
NCT05444894
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-04-02
First Post:
2022-06-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2025-11-10', 'mcpReleaseN': 11, 'releaseDate': '2025-10-27'}, {'resetDate': '2025-11-24', 'mcpReleaseN': 12, 'releaseDate': '2025-11-11'}, {'resetDate': '2025-12-18', 'mcpReleaseN': 13, 'releaseDate': '2025-12-04'}], 'estimatedResultsFirstSubmitDate': '2025-10-27'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 9}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2022-04-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-03-28', 'studyFirstSubmitDate': '2022-06-27', 'studyFirstSubmitQcDate': '2022-07-01', 'lastUpdatePostDateStruct': {'date': '2025-04-02', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-07-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days)', 'timeFrame': 'EDIT-301 infusion (Day 0) to 42 days post EDIT-301 infusion'}, {'measure': 'Frequency and severity of adverse events (AEs) (incidence of AEs and Grade 3 or higher serious adverse events, using National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] v.5.0)', 'timeFrame': 'Screening through up to 24 months post EDIT-301 infusion'}], 'secondaryOutcomes': [{'measure': 'Kinetics of HSPC engraftment', 'timeFrame': 'EDIT-301 infusion (Day 0) to first day in which 3 consecutive measurements obtained on different days demonstrate ANC ≥ 0.5 x 10^9/L up to 24 months post EDIT-301 infusion', 'description': 'Time to neutrophil engraftment'}, {'measure': 'Kinetics of HSPC engraftment', 'timeFrame': 'EDIT-301 infusion (Day 0) to first day of 3 consecutive measurements of platelets ≥ 50 x 10^9/L for at least 1 week following the last platelet transfusion and 10 days following thrombopoietin mimetics use up to 24 months post EDIT-301 infusion.', 'description': 'Time to platelet engraftment'}, {'measure': 'Incidence of transplant related mortality', 'timeFrame': 'EDIT-301 infusion (Day 0) through Day 100 post EDIT-301 infusion and from EDIT-301 infusion (Day 0) through 12 months post EDIT-301 infusion'}, {'measure': 'Incidence of all-cause mortality', 'timeFrame': 'Screening through up to 24 months post EDIT-301 infusion'}, {'measure': 'Proportion of alleles per participant with intended genetic modification present in peripheral blood over time', 'timeFrame': 'EDIT-301 infusion (Day 0) through up to 24 months post EDIT-301 infusion'}, {'measure': 'Proportion of alleles per participant with intended genetic modification present in bone marrow cells over time', 'timeFrame': 'EDIT-301 infusion (Day 0) through up to 24 months post EDIT-301 infusion'}, {'measure': 'Change in the fetal hemoglobin (HbF) concentration compared to baseline overtime', 'timeFrame': 'Baseline through up to 24 months post EDIT-301 infusion'}, {'measure': 'Change in the total hemoglobin concentration compared to baseline overtime', 'timeFrame': 'Baseline through up to 24 months post EDIT-301 infusion'}, {'measure': 'Proportion of participants with hemoglobin concentration ≥ 9 g/dL', 'timeFrame': 'EDIT-301 infusion (Day 0) through 3, 6, 12 months up to 24 months post EDIT-301 infusion'}, {'measure': 'Proportion of participants achieving the sustained transfusion reduction (TR) for at least 6 months and at least 12 months from 3 months post-EDIT-301 infusion', 'timeFrame': '3 months post EDIT-301 infusion through up to 24 months post EDIT-301 infusion'}, {'measure': 'Proportion of participants achieving the sustained transfusion independence (TI) for at least 6 months and, at least 12 months from 3 months post EDIT-301 infusion', 'timeFrame': '3 months through up to 24 months post EDIT-301 infusion'}, {'measure': 'Change in parameters of iron overload compared to baseline over time', 'timeFrame': 'Baseline through up to 24 months post EDIT-301 infusion'}, {'measure': 'Proportion of participants receiving iron chelation therapy over time', 'timeFrame': 'EDIT-301 infusion (Day 0) through up to 24 months post EDIT-301 infusion'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Beta-Thalassemia', 'Hemoglobinopathies', 'CRISPR-Cas 12a', 'Autologous CD34+'], 'conditions': ['Transfusion Dependent Beta Thalassemia', 'Hemoglobinopathies', 'Thalassemia Major', 'Thalassemia Intermedia']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia', 'detailedDescription': 'This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '35 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\nDiagnosis of Transfusion Dependent B-Thalassemia as defined by:\n\n* Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and\n* History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent\n* Clinically stable and eligible to undergo autologous HSCT\n* Karnofsky Performance Status ≥ 70\n\nKey Exclusion Criteria:\n\n* Available 10/10 human leukocyte antigen (HLA)-matched related donor\n* Prior HSCT or contraindications to autologous HSCT\n* Participants with associated a history of α-thalassemia and \\> 1 alpha chain deletion, or alpha multiplications as documented in medical records\n* Participants with a history of other inherited hemoglobinopathy or thalassemic mutation (Hb S, C, D or other) as documented in medical records\n* Prior receipt of gene therapy\n* Inadequate bone marrow function, as defined by white blood cell count of \\< 3 x 10\\^9/L or a platelet count \\< 100 x 10\\^9/L (without hypersplenism), per investigator judgement\n* Inadequate organ function\n* Advanced liver disease\n* Any prior or current malignancy, or immunodeficiency disorder,\n* Immediate family member with a known or suspected Familial Cancer Syndrome\n* Clinically significant and active bacterial, viral, fungal, or parasitic infection'}, 'identificationModule': {'nctId': 'NCT05444894', 'briefTitle': 'EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Editas Medicine, Inc.'}, 'officialTitle': 'A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)', 'orgStudyIdInfo': {'id': 'EM-301-BThal-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'EDIT-301', 'description': 'EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.', 'interventionNames': ['Genetic: EDIT-301']}], 'interventions': [{'name': 'EDIT-301', 'type': 'GENETIC', 'otherNames': ['renizgamglogene autogedtemcel', 'reni-cel'], 'description': 'Administered by intravenous infusion after myeloablative conditioning with busulfan.', 'armGroupLabels': ['EDIT-301']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': 'University of California San Francisco', 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '55410', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center - Department of Pediatrics', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '37203', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': "Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers", 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': 'M5G 2M9', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Princess Margaret Cancer Centre-University Health Network', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Editas Medicine, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}