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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Sickle Cell Disease Clinical Trial

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

Clinical Trial 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).

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05329649
Study type Interventional
Source Vertex Pharmaceuticals Incorporated
Contact Medical Information
Phone 617-341-6777
Email medicalinfo@vrtx.com
Status Recruiting
Phase Phase 3
Start date May 2, 2022
Completion date May 2026
View Details
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