A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
Vertex Pharmaceuticals Incorporated
Summary
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
Eligibility
- Age range
- 12–35 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: * Participants with TDT and SCD: * Eligible for autologous stem cell transplant as per investigator's judgment. * Participants with TDT: * Diagnosis of TDT as defined by: * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning * History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) t…
Interventions
- BiologicalCTX001
Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan
Locations (6)
- New York Presbyterian Hospital - Morgan Stanley Children's HospitalNew York, New York
- Levine Children's Hospital - HematologyCharlotte, North Carolina
- TriStar Medical Group Children's Specialists - Pediatric OncologyNashville, Tennessee
- University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical ImmunologyDüsseldorf
- IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e GenicaRome
- King Faisal Specialist Hospital & Research Centre - Riyadh - HematologyAl Mathar Ash Shamali