Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Recruiting now · Phase 3
Conditions studied: Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
In brief
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.
Key facts
- Study ID
- NCT05477563
- Run by
- Vertex Pharmaceuticals Incorporated
- People needed
- 26
- Starts
- 2022-08-02
- Expected to finish
- 2027-06-09
- Last updated by the study team
- 2026-03-23
Who can join
Age: 12 and older, up to 35. Sex: any. Healthy volunteers: not accepted.
Where it is running
- New York Presbyterian Hospital - Morgan Stanley Children's Hospital — New York, New York, United States (enrolling)
- Levine Children's Hospital - Hematology — Charlotte, North Carolina, United States (enrolling)
- TriStar Medical Group Children's Specialists - Pediatric Oncology — Nashville, Tennessee, United States (enrolling)
- University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology — Düsseldorf, Germany (enrolling)
- IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica — Rome, Italy (enrolling)
- King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology — Al Mathar Ash Shamali, Saudi Arabia (enrolling)
Full record on ClinicalTrials.gov
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