A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
← catalyst calendarSponsored by Vertex Pharmaceuticals Incorporated (industry) · VRTX — their whole pipeline →. With CRISPR Therapeutics.
Phase
Phase 2/3
Status
Completed
Study type
Interventional
Design
Na · None · Treatment
Enrollment
63actual
Sites
17
Countries
Belgium, Canada, France +4
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 | 2018-11-27 | actual | When the study began enrolling |
| Primary completion | 2025-07-07 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-07-07 | actual | The whole study's end, after follow-up |
| First posted | 2018-11-19 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-08-11 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Sickle Cell Disease
- Hematological Diseases
- Hemoglobinopathies
Intervention
- Biological: CTX001 — also filed as Exagamglogene autotemcel, Exa-cel
Primary outcomes
what the primary-completion date above is the date OFProportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12)
measured From 60 days after last RBC transfusion up to 2 years after CTX001 infusion
Proportion of subjects with engraftment (first day of three consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days)
measured Within 42 days after CTX001 infusion
Time to engraftment
measured From CTX001 infusion up to 2 years after CTX001 infusion
Frequency and severity of collected adverse events (AEs)
measured From screening to 2 years after CTX001 infusion
Incidence of transplant-related mortality (TRM) within 100 days after CTX001 infusion
measured Within 100 days after CTX001 infusion
Incidence of TRM within 1 year after CTX001 infusion
measured Within 1 year after CTX001 infusion
All-cause mortality
measured 2 years after mobilization
Publications
- PMID 40857358 — linked by the registry
- PMID 38866240 — linked by the registry
- PMID 38661449 — linked by the registry
- PMID 36975201 — linked by the registry
- PMID 36860093 — linked by the registry
- PMID 36123234 — linked by the registry
- PMID 34175041 — linked by the registry
- PMID 33283989 — linked by the registry
- PMID 32873924 — 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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