Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
← catalyst calendarSponsored by Rocket Pharmaceuticals Inc. (industry) · RCKT — their whole pipeline →.
Phase
Phase 2
Status
Unknown status
Study type
Interventional
Design
Na · None · Treatment
Enrollment
7actual
Sites
2
Countries
Spain, United Kingdom
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 | 2019-11-28 | actual | When the study began enrolling |
| Primary completion | Feb 2025 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Feb 2025 | estimated | The whole study's end, after follow-up |
| First posted | 2019-08-28 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2024-05-22 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Fanconi Anemia Complementation Group A
Intervention
- Biological: RP-L102
Primary outcome
what the primary-completion date above is the date OFPhenotypic correction of bone marrow colony forming units after infusion of RP-L102
measured 3 years
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