A Long-term Follow-up Study in Participants Who Received CTX001

PHASE3Enrolling by invitationINTERVENTIONAL
Enrollment

160

Participants

Timeline

Start Date

January 20, 2021

Primary Completion Date

September 30, 2039

Study Completion Date

September 30, 2039

Conditions
Beta-ThalassemiaThalassemiaSickle Cell DiseaseHematologic DiseasesHemoglobinopathiesGenetic Diseases, InbornSickle Cell Anemia
Interventions
BIOLOGICAL

CTX001

CTX001 infusion.

Trial Locations (19)

10032

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

Columbia University Medical Center, New York

19104

Children's Hospital of Philadelphia, Philadelphia

28203

Atrium Health Levine Children's Hospital, Charlotte

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

78229

Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital, San Antonio

94304

Lucile Packard Children's Hospital, Palo Alto

Unknown

Hopital Universitaire des Enfants Reine Fabiola (HUDERF), Brussels

The Hospital for Sick Children, Toronto

Toronto General Hospital, University Health Network, Toronto

St. Paul's Hospital, Vancouver

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

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

University Hospital Tuebingen, Tübingen

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