Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
← catalyst calendarNCT04437771 · readout in 2,708 d
Sponsored by Rocket Pharmaceuticals Inc. (industry) · RCKT — their whole pipeline →.
Phase
—
Status
Active, not recruiting
Study type
Observational
Enrollment
9estimated
Sites
1
Country
Spain
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 | 2020-06-01 | actual | When the study began enrolling |
| Primary completion | 2034-01-17 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2034-01-17 | estimated | The whole study's end, after follow-up |
| First posted | 2020-06-18 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-04-13 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Fanconi Anemia Complementation Group A
- Fanconi Anemia
Intervention
- Other: Safety and efficacy assessments
Primary outcomes
what the primary-completion date above is the date OFMonitor long term safety of patients through blood laboratory evaluations and general health status
measured 15 years post-drug product infusion
Long term genetic correction assessed in bone marrow and blood
measured 15 years post-drug product infusion
Replication competent lentivirus (RCL)
measured 15 years post-drug product infusion
Insertion site analysis in blood
measured 15 years post-drug product infusion
Phenotypic correction
measured 15 years post-drug product infusion
Assessment for Malignancies
measured 15 years post-drug product infusion
Hematologic stabilization
measured 15 years post-drug product infusion
Publications
- PMID 39642902 — 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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