ClinicaliQ Trial Snapshot
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) — Active Not Recruiting • Phase III • NCT05329649.
- Gene-edited stem cell therapy significantly improves severe sickle cell disease in children who cannot tolerate standard treatment.
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What This Trial Is Studying
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). Conditions: Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases Interventions: CTX001 Lead Sponsor: Vertex Pharmaceuticals Incorporated Planned Enrollment: 13 participants