A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

PHASE2/PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

59

Participants

Timeline

Start Date

September 14, 2018

Primary Completion Date

December 8, 2025

Study Completion Date

December 8, 2025

Conditions
Beta-ThalassemiaThalassemiaGenetic Diseases, InbornHematologic DiseasesHemoglobinopathies
Interventions
BIOLOGICAL

CTX001

Administered by IV infusion following myeloablative conditioning with busulfan

Trial Locations (14)

10032

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

Columbia University Medical Center, 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

60611

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

94304

Lucile Packard Children's Hospital, Palo Alto

Unknown

The Hospital for Sick Children, Toronto

British Columbia Children's Hospital, Vancouver

Universitätsklinikum Düsseldorf Hospital Duesseldorf, Düsseldorf

Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Regensburg

University Hospital Tübingen, Tübingen

Ospedale Pediatrico Bambino Gesù, IRCCS, Rome

Imperial College Healthcare NHS Trust, Hammersmith Hospital, London

University College London Hospitals NHS Foundation Trust, London

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

CRISPR Therapeutics

INDUSTRY

lead

Vertex Pharmaceuticals Incorporated

INDUSTRY