A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
- Conditions
- HemoglobinopathiesSickle Cell DiseaseTransfusion-dependent Beta-Thalassemia
- Interventions
- Other: Safety and efficacy assessments
- Registration Number
- NCT06363760
- Lead Sponsor
- Editas Medicine, Inc.
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 54
- Participant must have received an EDIT-301 infusion as part of a clinical study.
- 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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description All participants Safety and efficacy assessments All participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001)
- Primary Outcome Measures
Name Time Method Adverse events of special interest (AESI) up to 15 years post EDIT-301 infusion Adverse events of interest (AEI) up to 15 years post EDIT-301 infusion Adverse events (AEs) related to EDIT-301 up to 15 years post EDIT-301 infusion Serious adverse events (SAEs) up to 15 years post EDIT-301 infusion All-cause mortality up to 15 years post EDIT-301 infusion
- Secondary Outcome Measures
Name Time Method SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time up to 15 years post EDIT-301 infusion HbF concentration (g/dL) over time for TDT participants up to 15 years post EDIT-301 infusion Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion up to 15 years post EDIT-301 infusion Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion up to 15 years post EDIT-301 infusion Complete Blood Count (CBC) red cell indices assay over time for SCD participants up to 15 years post EDIT-301 infusion 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. up to 15 years post EDIT-301 infusion Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants up to 15 years post EDIT-301 infusion 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. up to 15 years post EDIT-301 infusion Proportion of TDT participants with sustained transfusion reduction (TR) over time up to 15 years post EDIT-301 infusion Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants up to 15 years post EDIT-301 infusion SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time up to 15 years post EDIT-301 infusion HbF and HbS concentration (g/dL) over time for SCD participants up to 15 years post EDIT-301 infusion Proportion of TDT participants receiving iron chelation therapy over time. up to 15 years post EDIT-301 infusion Proportion of TDT participants with sustained transfusion independence (TI) over time up to 15 years post EDIT-301 infusion Average HbF (pg) per RBC over time for SCD participants up to 15 years post EDIT-301 infusion SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time up to 15 years post EDIT-301 infusion Average HbF (pg) per F-cell over time for SCD participants up to 15 years post EDIT-301 infusion Iron overload of TDT participants up to 15 years post EDIT-301 infusion 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.
Trial Locations
- Locations (18)
Smilow Cancer Hospital
🇺🇸New Haven, Connecticut, United States
Johns Hopkins All Children's Hospital
🇺🇸Saint Petersburg, Florida, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Columbia University Medical Center - Department of Pediatrics
🇺🇸New York, New York, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers
🇺🇸Nashville, Tennessee, United States
Texas Oncology - Baylor Charles A. Sammons Cancer Center
🇺🇸Dallas, Texas, United States
Cook Children's
🇺🇸Fort Worth, Texas, United States
Ottawa Hospital Research Institute
🇨🇦Ottawa, Ontario, Canada
Atrium Health
🇺🇸Charlotte, North Carolina, United States
Princess Margaret Cancer Centre-University Health Network
🇨🇦Toronto, Ontario, Canada
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States