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

Part of paid clinical trials in Palo Alto, California.

Sponsor
Rocket Pharmaceuticals Inc.
Study ID
NCT07527975
Status
Enrolling By Invitation

Conditions

  • Fanconi Anemia

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • RP-L102 — BIOLOGICAL
    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

Study Details

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

Key Dates

First listed
Apr 14, 2026
Start date
Feb 1, 2022
Status verified
Apr 2026
Primary completion
May 31, 2038
Completion
May 31, 2038

Study Design

Enrollment
14 participants (estimated)

Arms

  • Arm: Subjects that received RP-L102 on the RP-L102-0418, RP-L102-0118 and RP-L102-0319 parent studies
    Subjects that received RP-L102 on the RP-L102-0418, RP-L102-0118 and RP-L102-0319 parent studies and either completed the study or discontinued early.

Primary Outcome Measure

Survival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118). [ Time Frame: From infusion in parent study to 15-years post-infusion. ]

Locations (1)

FacilityCityStateZIPSite coordinators
Lucille Packard Children's Hospital, Stanford UniversityPalo AltoCalifornia94305-

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Lucille Packard Children's Hospital, Stanford University· Palo Alto, CA

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