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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All participants | All participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Safety and efficacy assessments | Other | Assessed throughout the duration of the study. |
|
| Measure | Description | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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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)
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | United States | ||
| Children's Hospital Colorado |
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| 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 |
| 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 |
| Average HbF (pg) per F-cell over time for SCD participants | 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 | 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. | 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 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Smilow Cancer Hospital | New Haven | Connecticut | 06511 | United States |
| Johns Hopkins All Children's Hospital | St. Petersburg | Florida | 33701 | United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55410 | United States |
| Columbia University Medical Center - Department of Pediatrics | New York | New York | 10032 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Atrium Health | Charlotte | North Carolina | 28204 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers | Nashville | Tennessee | 37203 | United States |
| Texas Oncology - Baylor Charles A. Sammons Cancer Center | Dallas | Texas | 75246 | United States |
| Cook Children's | Fort Worth | Texas | 76104 | United States |
| Ottawa Hospital Research Institute | Ottawa | Ontario | K1H 8L6 | Canada |
| Princess Margaret Cancer Centre-University Health Network | Toronto | Ontario | M5G 2M9 | Canada |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D006453 | Hemoglobinopathies |
| D017086 | beta-Thalassemia |
| D030342 | Genetic Diseases, Inborn |
| D006402 | Hematologic Diseases |
| D000740 | Anemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013789 | Thalassemia |
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| ID | Term |
|---|---|
| D012449 | Safety |
| ID | Term |
|---|---|
| D000056 | Accident Prevention |
| D000059 | Accidents |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
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