A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
Status: Active_not_recruiting
Location: See all (7) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 3
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).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 11
Healthy Volunteers: f
View:
• Diagnosis of severe SCD as defined by:
• Documented SCD genotypes
• History of at least two severe VOCs events per year for the previous two years prior to enrollment
• Hydroxyurea (HU) failure unless HU intolerant
• Eligible for autologous stem cell transplant as per investigators judgment
Locations
United States
North Carolina
Levine Children's Hospital - Hematology
Charlotte
Pennsylvania
The Children's Hospital of Philadelphia - Hematology
Philadelphia
Tennessee
St. Jude Children's Research Hospital
Memphis
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville
Other Locations
Germany
University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf
Italy
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome
United Kingdom
St.Mary's Hospital - Haematology Dept
London
Time Frame
Start Date: 2022-05-02
Completion Date: 2026-05-31
Participants
Target number of participants: 13
Treatments
Experimental: CTX001
CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive single infusion of CTX001 through central venous catheter.
Related Therapeutic Areas
Sponsors
Leads: Vertex Pharmaceuticals Incorporated
Collaborators: CRISPR Therapeutics