NCT05329649View on ClinicalTrials.gov

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

PHASE3RecruitingINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

May 2, 2022

Primary Completion Date

May 31, 2026

Study Completion Date

May 31, 2026

Conditions
Sickle Cell DiseaseHydroxyurea FailureHydroxyurea IntoleranceHemoglobinopathiesHematological Diseases
Interventions
BIOLOGICAL

CTX001

Administered by intravenous infusion following myeloablative conditioning with busulfan.

Trial Locations (7)

19104

RECRUITING

The Children's Hospital of Philadelphia - Hematology, Philadelphia

28203

RECRUITING

Levine Children's Hospital - Hematology, Charlotte

37203

RECRUITING

TriStar Medical Group Children's Specialists - Pediatric Oncology, Nashville

38105

RECRUITING

St. Jude Children's Research Hospital, Memphis

Unknown

RECRUITING

University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology, Düsseldorf

RECRUITING

IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica, Rome

RECRUITING

St.Mary's Hospital - Haematology Dept, London

Sponsors

Collaborators (1)

CRISPR Therapeutics
All Listed Sponsors
collaborator

CRISPR Therapeutics

INDUSTRY

lead

Vertex Pharmaceuticals Incorporated

INDUSTRY

NCT05329649 - Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | Biotech Hunter | Biotech Hunter