Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
← catalyst calendarNCT05477563 · readout in 293 d
Sponsored by Vertex Pharmaceuticals Incorporated (industry) · VRTX — their whole pipeline →.
Phase
Phase 3
Status
Recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
26estimated
Sites
6
Countries
Germany, Italy, Saudi Arabia +1
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 | 2022-08-02 | actual | When the study began enrolling |
| Primary completion | 2027-06-09 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2027-06-09 | estimated | The whole study's end, after follow-up |
| First posted | 2022-07-28 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-03-23 | 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
- Hematologic Diseases
- Genetic Diseases, Inborn
- Hemoglobinopathies
- Sickle Cell Disease
- Sickle Cell Anemia
Intervention
- Biological: CTX001 — also filed as Exagamglogene autotemcel, Exa-cel
Primary outcomes
what the primary-completion date above is the date OFFetal Hemoglobin (HbF) Concentration Over Time
measured Up to 12 Months After CTX001 Infusion
Total Hemoglobin (Hb) Concentration Over Time
measured Up to 12 Months After CTX001 Infusion
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