A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Study type

Interventional

Primary purpose

Treatment

Allocation

Not applicable

Intervention model

Single group

Masking

None

Arms

1

Interventions

• CTX001 Biological

• Beta-Thalassemia
• Thalassemia
• Genetic Diseases, Inborn
• Hematologic Diseases
• Hemoglobinopathies

Primary outcome

Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12) (From 60 days after last RBC transfusion up to 24 months post-CTX001 infusion]); Proportion of participants with engraftment (first day of 3 consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days) (Within 42 days after CTX001 infusion); Time to neutrophil and platelet engraftment (Days post-infusion to engraftment); Frequency and severity of collected adverse events (AEs) (Signing of informed consent through Month 24 visit); Incidence of transplant-related mortality (TRM) (Baseline (pre-transfusion) to 100 days and 1 year post-CTX001 infusion); All-cause mortality (Signing of informed consent through Month 24 visit)

• CTX001

• NCT03655678 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read 25 Oct 2026

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.