Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
← catalyst calendarNCT05356195 · readout in 452 d
Sponsored by Vertex Pharmaceuticals Incorporated (industry) · VRTX — their whole pipeline →. With CRISPR Therapeutics.
Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
16actual
Sites
6
Countries
Canada, Germany, Italy +2
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-05-03 | actual | When the study began enrolling |
| Primary completion | 2027-11-14 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2027-11-14 | estimated | The whole study's end, after follow-up |
| First posted | 2022-05-02 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-01 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Beta-Thalassemia
- Thalassemia
- Genetic Diseases, Inborn
- Hematologic Diseases
- Hemoglobinopathies
Intervention
- Biological: CTX001 — also filed as Exagamglogene autotemcel, Exa-cel
Primary outcome
what the primary-completion date above is the date OFProportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)
measured Up to 24 Months After CTX001 Infusion
Publications
- PMID 42274009 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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