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

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)	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, thalassemia	Active, not recruiting	Phase 3	2026-07-01		NCT05356195

2 results · Page 1 of 1

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Organizations	Linked trials
CRISPR Therapeutics	6
Vertex Pharmaceuticals Incorporated	37

6 results · Page 1 of 1

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Research sites	City	Country	Linked trials
Great Ormond Street Hospital for Children	London	United Kingdom	15
Hospital for Sick Children - Hematology	Toronto	Canada	2
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica	Rome	Italy	4
St.Mary's Hospital - Children's Clinical Research Facility	London	United Kingdom	1
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