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Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102

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NCT07527975 · readout ≤ 4,303 d

Sponsored by Rocket Pharmaceuticals Inc. (industry) · RCKT — their whole pipeline →.

Phase
Status
Enrolling by invitation
Study type
Observational
Enrollment
14estimated
Sites
3
Countries
Spain, United Kingdom, United States

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
DateFiledBasisWhat it is
Start2022-02-01actualWhen the study began enrolling
Primary completionMay 2038estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completionMay 2038estimatedThe whole study's end, after follow-up
First posted2026-04-14actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-04-14actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

  • Fanconi Anemia

Intervention

  • Biological: RP-L102

Primary outcomes

what the primary-completion date above is the date OF
Survival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
measured From infusion in parent study to 15-years post-infusion.
Long term safety
measured From infusion in parent study to 15-years post-infusion.
Long-term persistence of the therapeutic LV (provirus) in hematopoietic cells in the bone marrow (BM) and blood.
measured From infusion in parent study to 15-years post-infusion.
Long-term clonality patterns.
measured From infusion in parent study to 15-years post-infusion.
Replication-competent lentivirus (RCL) in serum and peripheral blood cells.
measured From infusion in parent study to 15-years post-infusion.
Long-term stability and normalization of blood counts.
measured From infusion in parent study to 15-years post-infusion.
Phenotypic correction of BM and peripheral blood cells
measured From infusion in parent study to 15-years post-infusion.
Incidence of hematologic malignancies and solid organ tumors.
measured From infusion in parent study to 15-years post-infusion.

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