NCT05477563View on ClinicalTrials.gov

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

PHASE3RecruitingINTERVENTIONAL
Enrollment

26

Participants

Timeline

Start Date

August 2, 2022

Primary Completion Date

June 9, 2027

Study Completion Date

June 9, 2027

Conditions
Beta-ThalassemiaThalassemiaHematologic DiseasesGenetic Diseases, InbornHemoglobinopathiesSickle Cell DiseaseSickle Cell Anemia
Interventions
BIOLOGICAL

CTX001

Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan

Trial Locations (6)

10032

RECRUITING

Columbia University Medical Center, New York

28203

RECRUITING

Atrium Health Levine Children's Hospital, Charlotte

37203

RECRUITING

TriStar Medical Group Children's Specialist, Nashville

Unknown

RECRUITING

Universitätsklinikum Düsseldorf Hospital Duesseldorf, Düsseldorf

ACTIVE_NOT_RECRUITING

Ospedale Pediatrico Bambino Gesù, IRCCS, Rome

RECRUITING

King Faisal Specialist Hospital and Research Centre, Al Mathar Ash Shamali

All Listed Sponsors
lead

Vertex Pharmaceuticals Incorporated

INDUSTRY