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

Enrolling by invitation

Conditions studied: Fanconi Anemia

In brief

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

Key facts

Study ID

NCT07527975

Run by

Rocket Pharmaceuticals Inc.

People needed

14

Starts

2022-02-01

Expected to finish

2038-05-01

Last updated by the study team

2026-04-14

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Enrolled in one of the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
• Received an autologous infusion of CD34+ enriched cells transduced ex vivo with LV vector carrying the FANCA gene, PGK-FANCA-WPRE (RP-L102), in the parent studies.
• Able to adhere to the study visit schedule and other protocol requirements.
• Has provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements.

You may not qualify if…

• There are no criteria for exclusion in this study.

Where it is running

• Lucille Packard Children's Hospital, Stanford University — Palo Alto, California, United States
• Hospital Infantil Universitario Niño Jesús — Madrid, Spain
• University College London Great Ormond Street Institute of Child Health (GOSH) — London, United Kingdom

Full record on ClinicalTrials.gov

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