Hemoglobinopathies, Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
Conditions
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
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.
Interventions
Assessed throughout the duration of the study.
Sponsors
Editas Medicine, Inc.
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 |
| All-cause mortality | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time | up to 15 years post EDIT-301 infusion | — |
| SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time | 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 | — |
| Average HbF (pg) per RBC over time for SCD participants | 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 | — |
| Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion | 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 | — |
| 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 | — |
| 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 | — |
| HbF concentration (g/dL) over time for TDT 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. |
| Proportion of TDT participants receiving iron chelation therapy 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 | — |
| Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion | 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 | — |
| Proportion of TDT participants with sustained transfusion independence (TI) over time | 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 | — |
Countries
Canada, United States
Outcome results
None listed