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

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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