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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
Sponsor: Vertex Pharmaceuticals Incorporated
Summary
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).
Official title: A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
Key Details
Gender
All
Age Range
2 Years - 11 Years
Study Type
INTERVENTIONAL
Enrollment
13
Start Date
2022-05-02
Completion Date
2026-05-31
Last Updated
2026-03-09
Healthy Volunteers
No
Conditions
Interventions
CTX001
Administered by intravenous infusion following myeloablative conditioning with busulfan.
Locations (7)
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