NCT05356195View on ClinicalTrials.gov

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

PHASE3RecruitingINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

May 3, 2022

Primary Completion Date

May 31, 2026

Study Completion Date

May 31, 2026

Conditions
Beta-ThalassemiaThalassemiaGenetic Diseases, InbornHematologic DiseasesHemoglobinopathies
Interventions
BIOLOGICAL

CTX001

Administered by intravenous infusion following myeloablative conditioning with busulfan.

Trial Locations (6)

37203

RECRUITING

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

Unknown

RECRUITING

Hospital for Sick Children - Hematology, Toronto

RECRUITING

University Hospital Dusseldorf - 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

Great Ormond Street Hospital for Children, London

RECRUITING

St.Mary's Hospital - Children's Clinical Research Facility, London

Sponsors

Collaborators (1)

CRISPR Therapeutics
All Listed Sponsors
collaborator

CRISPR Therapeutics

INDUSTRY

lead

Vertex Pharmaceuticals Incorporated

INDUSTRY

NCT05356195 - Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | Biotech Hunter | Biotech Hunter