Found 7 matching trials
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
PHASE3This is a single-dose, open-label study in pediatric participants with seve...
Status
Active Not Recruiting
Location
Levine Children's Hospital - Hematology
A Long-term Follow-up Study in Participants Who Received CTX001
PHASE3This is a multi-site, open- label rollover study to evaluate the long-term ...
Status
Enrolling By Invitation
Location
Lucile Packard Children's Hospital
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
PHASE3This is a single-dose, open-label study in pediatric participants with TDT....
Status
Active Not Recruiting
Location
TriStar Medical Group Children's Specialists - Pediatric Oncology
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
PHASE3This is a single-dose, open-label study in participants with transfusion-de...
Location
New York Presbyterian Hospital - Morgan Stanley Children's Hospital
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
PHASE3The purpose of the study is to evaluate the efficacy and safety of CTX001 (...
Location
Multiple Locations
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
PHASE2This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study...
Location
Lucile Packard Children's Hospital of Stanford University
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
PHASE2This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study...
Location
Lucile Packard Children's Hospital