NCT07527975

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

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 14 (estimated)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102

Detailed description

Following the end of participation in Study(RP-L102-0418, RP-L102-0319, RP-L102-0118), patients will be offered enrollment into this LTFU protocol. Patients will be followed for up to 15 years following the RP-L102 infusion in the parent study, until the patient dies, withdraws consent, or is lost to follow-up (whichever occurs first). For all follow-up visits, remote evaluation facilitated by local health care providers (with blood sample shipment to relevant laboratory facilities) is permitted; however, visits to the study center are required for up to 2 years post- RP-L102 infusion. Study center visits are encouraged when feasible, especially in years 2 through 5 following gene therapy administration. Blood samples will be archived and tested when clinically or scientifically indicated, as in the event of development of a second malignancy.

Conditions

• Fanconi Anemia

Interventions

Type	Name	Description
BIOLOGICAL	RP-L102	CD34+ enriched cells from subjects with Fanconi anemia subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the FANCA gene, PGK-FANCA-WPRE

Timeline

• Start date: 2022-02-01
• Primary completion: 2038-05-01
• Completion: 2038-05-01
• First posted: 2026-04-14
• Last updated: 2026-04-14

Locations

3 sites across 3 countries: United States, Spain, United Kingdom

Regulatory

• FDA-regulated drug study