Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
Running, not enrolling · Phase 3
Conditions studied: Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
In brief
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
Key facts
- Study ID
- NCT05329649
- Run by
- Vertex Pharmaceuticals Incorporated
- People needed
- 13
- Starts
- 2022-05-02
- Expected to finish
- 2027-06-06
- Last updated by the study team
- 2026-07-01
Who can join
Age: 2 and older, up to 11. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Levine Children's Hospital - Hematology — Charlotte, North Carolina, United States
- The Children's Hospital of Philadelphia - Hematology — Philadelphia, Pennsylvania, United States
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States
- TriStar Medical Group Children's Specialists - Pediatric Oncology — Nashville, Tennessee, United States
- University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology — Düsseldorf, Germany
- IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica — Rome, Italy
- St.Mary's Hospital - Haematology Dept — London, United Kingdom
Full record on ClinicalTrials.gov
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