Ignite Creation Date:
2025-12-25 @ 2:17 AM
Ignite Modification Date:
2025-12-30 @ 3:23 PM
Study NCT ID:
NCT06363760
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-04-02
First Post:
2024-04-09
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D017086', 'term': 'beta-Thalassemia'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}], 'ancestors': [{'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013789', 'term': 'Thalassemia'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D012449', 'term': 'Safety'}], 'ancestors': [{'id': 'D000056', 'term': 'Accident Prevention'}, {'id': 'D000059', 'term': 'Accidents'}, {'id': 'D011634', 'term': 'Public Health'}, {'id': 'D004778', 'term': 'Environment and Public Health'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 54}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2024-06-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2040-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-03-28', 'studyFirstSubmitDate': '2024-04-09', 'studyFirstSubmitQcDate': '2024-04-09', 'lastUpdatePostDateStruct': {'date': '2025-04-02', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-04-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2040-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Adverse events of special interest (AESI)', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Adverse events of interest (AEI)', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'All-cause mortality', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Adverse events (AEs) related to EDIT-301', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Serious adverse events (SAEs)', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}], 'secondaryOutcomes': [{'measure': 'Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Proportion of TDT participants with sustained transfusion independence (TI) over time', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Proportion of TDT participants with sustained transfusion reduction (TR) over time', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Average HbF (pg) per RBC over time for SCD participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Complete Blood Count (CBC) red cell indices assay over time for SCD participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Average HbF (pg) per F-cell over time for SCD participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'HbF and HbS concentration (g/dL) over time for SCD participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g/dL of all Hb variants, excluding HbA) over time for TDT participants.', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'HbF concentration (g/dL) over time for TDT participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}, {'measure': 'Iron overload of TDT participants', 'timeFrame': 'up to 15 years post EDIT-301 infusion', 'description': 'As measured by a) Liver iron concentration (LIC) as assessed by R2\\* magnetic resonance imaging (MRI) for the first 3 years in this study; b) Cardiac iron concentration (CIC) as assessed by T2\\* MRI for the first 3 years of this study; c) Serum ferritin levels over time.'}, {'measure': 'Proportion of TDT participants receiving iron chelation therapy over time.', 'timeFrame': 'up to 15 years post EDIT-301 infusion'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Sickle Cell Disease', 'Sickle Cell Anemia', 'Sickling Disorder Due to Hemoglobin S', 'Beta-Thalassemia', 'Thalassemia Major', 'Thalassemia Intermedia', 'Hemoglobinopathies', 'CRISPR-Cas12a', 'Autologous CD34+', 'Genetic Diseases, Inborn', 'Hematologic Diseases', 'Anemia', 'Anemia, Hemolytic, Congenital', 'Anemia, Hemolytic'], 'conditions': ['Sickle Cell Disease', 'Transfusion-dependent Beta-Thalassemia', 'Hemoglobinopathies']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.', 'detailedDescription': 'This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '12 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'The study population will be subjects currently enrolled in any one of the two parent studies ( EM-SCD-301-001 or EM-301-BThal-001)', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participant must have received an EDIT-301 infusion as part of a clinical study.\n* Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.\n\nExclusion Criteria:\n\n* Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.'}, 'identificationModule': {'nctId': 'NCT06363760', 'briefTitle': 'A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301', 'organization': {'class': 'INDUSTRY', 'fullName': 'Editas Medicine, Inc.'}, 'officialTitle': 'A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301', 'orgStudyIdInfo': {'id': 'EDIT-301-LTFU-001'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'All participants', 'description': 'All participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001)', 'interventionNames': ['Other: Safety and efficacy assessments']}], 'interventions': [{'name': 'Safety and efficacy assessments', 'type': 'OTHER', 'description': 'Assessed throughout the duration of the study.', 'armGroupLabels': ['All participants']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "UCSF Benioff Children's Hospital", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '06511', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Smilow Cancer Hospital', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '33701', 'city': 'St. Petersburg', 'state': 'Florida', 'country': 'United States', 'facility': "Johns Hopkins All Children's Hospital", 'geoPoint': {'lat': 27.77086, 'lon': -82.67927}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Children's Healthcare of Atlanta", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'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': '28204', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Atrium Health', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'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': '75246', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Texas Oncology - Baylor Charles A. Sammons Cancer Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '76104', 'city': 'Fort Worth', 'state': 'Texas', 'country': 'United States', 'facility': "Cook Children's", 'geoPoint': {'lat': 32.72541, 'lon': -97.32085}}, {'zip': 'K1H 8L6', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Ottawa Hospital Research Institute', 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'zip': 'M5G 2M9', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Princess Margaret Cancer Centre-University Health Network', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Editas Medicine, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}