Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Recruiting · NCT05477563 · Registry updated 2026-03-23Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, beta thalassemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease, thalassemia | Recruiting | Phase 3 | 2026-03-23 | NCT05477563 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Vertex Pharmaceuticals Incorporated | 37 |
6 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica | Rome | Italy | 4 |
| King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology | Al Mathar Ash Shamali | Saudi Arabia | 1 |
| Levine Children's Hospital - Hematology | Charlotte | United States | 3 |
| New York Presbyterian Hospital - Morgan Stanley Children's Hospital | New York | United States | 2 |
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville | United States | 4 |
| University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology | Düsseldorf | Germany | 2 |