A Long-term Follow-up Study in Participants Who Received CTX001
← catalyst calendarNCT04208529 · readout in 4,790 d
Sponsored by Vertex Pharmaceuticals Incorporated (industry) · VRTX — their whole pipeline →. With CRISPR Therapeutics.
Phase
Phase 3
Status
Enrolling by invitation
Study type
Interventional
Design
Na · None · Other
Enrollment
160estimated
Sites
20
Countries
Belgium, Canada, Germany +3
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 | 2021-01-20 | actual | When the study began enrolling |
| Primary completion | 2039-09-30 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2039-09-30 | estimated | The whole study's end, after follow-up |
| First posted | 2019-12-23 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-08-11 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Beta-Thalassemia
- Thalassemia
- Sickle Cell Disease
- Hematologic Diseases
- Hemoglobinopathies
- Genetic Diseases, Inborn
- Sickle Cell Anemia
Intervention
- Biological: CTX001 — also filed as Exagamglogene autotemcel, Exa-cel
Primary outcomes
what the primary-completion date above is the date OFNew malignancies
measured Signing of informed consent up to 15 years post CTX001 infusion
New or worsening hematologic disorders
measured Signing of informed consent up to 15 years post CTX001 infusion
All-cause mortality
measured Signing of informed consent up to 15 years post CTX001 infusion
Serious adverse events (SAEs)
measured Signing of informed consent up to 15 years post CTX001 infusion
CTX001-related adverse events (AEs)
measured Signing of informed consent up to 15 years post CTX001 infusion
Publications
- PMID 42252696 — linked by the registry
- PMID 40862696 — linked by the registry
- PMID 40857358 — 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.
Permalink · VRTX's whole pipeline · Catalyst calendar · Every registered study · What changed