Gene Editing For Sickle Cell Disease
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary O
| Condition(s) | Sickle Cell Disease |
|---|---|
| Status | Recruiting |
| Phase | Phase 1 |
| Study type | Interventional |
| Summary | This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD. Secondary Objective * To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD. |
| Who can participate | Inclusion Criteria: * Age ≥18 years and ≤24.9 years. * Patients with SCD (Hb SS, Hb SB0 and Hb SB+ genotype) who have experienced EITHER (a) 2 or more SCD-related vaso-occlusive events (acute pain events, acute chest syndrome, priapism and splenic sequestration) per year in the 2-year period before screening, OR (b) administration of regular red blood cell (RBC) transfusions (≥8 transfusions in the 12 months preceding enrollment) EXCEPT if the RBC transfusions are being administered for primary or secondary stroke prevention and, in the opinion of the treating hematologist, cannot be safely discontinued after infusion of the gene modified drug product. * Failure, intolerance, or refusal of hydroxyurea therapy. * Patients must be eligible for autologous stem cell transplant as per investiga |
| Ages | 18 Years to 24 Years |
| Sex | All |
| Lead sponsor | St. Jude Children's Research Hospital |
| Locations | Memphis, Tennessee, United States |
| Start date | 2025-03-21 |
| NCT ID | NCT06506461 |
| Official listing | https://clinicaltrials.gov/study/NCT06506461 |