A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

PHASE2/PHASE3CompletedINTERVENTIONAL
Enrollment

63

Participants

Timeline

Start Date

November 27, 2018

Primary Completion Date

July 7, 2025

Study Completion Date

July 7, 2025

Conditions
Sickle Cell DiseaseHematological DiseasesHemoglobinopathies
Interventions
BIOLOGICAL

CTX001

Administered by IV infusion following myeloablative conditioning with busulfan.

Trial Locations (17)

10032

Columbia University Medical Center (21+ years), New York

Columbia University Medical Center (≤21 years), New York

19104

Children's Hospital of Philadelphia, Philadelphia

37203

The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, Nashville

38105

St. Jude Children's Research Hospital, Memphis

60611

Ann & Robert Lurie Children's Hospital of Chicago, Chicago

60612

University of Illinois at Chicago Hospitals and Health Systems, Chicago

78229

Methodist Children's Hospital/Texas Transplant Institute, San Antonio

94304

Lucile Packard Children's Hospital of Stanford University, Palo Alto

Unknown

Hopital Universitaire des Enfants Reine Fabiola (HUDERF), Brussels

The Hospital for Sick Children, Toronto

Hopital Necker Enfants Malades, Paris

University Hospital Duesseldorf, Düsseldorf

Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation, Regensburg

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

Imperial College Healthcare NHS Trust, Hammersmith Hospital, London

Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building, London

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

CRISPR Therapeutics

INDUSTRY

lead

Vertex Pharmaceuticals Incorporated

INDUSTRY