Clinical Trial

Gene Therapy for Fanconi Anemia, Complementation Group A

Active, Not Recruiting Phase 2
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Record status
This record was last updated December 22, 2025 (before its estimated May 5, 2026 completion). Its status may not reflect the trial's current state.
Summary
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2020-01-28; most recent amendment 2025-12-18.
Status change: Recruiting → Active, Not Recruiting 2024-04-08
Trial Details
NCT Number NCT04248439
Lead Sponsor Rocket Pharmaceuticals Inc.
Conditions Fanconi Anemia Complementation Group A
Enrollment 5 participants
Start Date 2020-07-15
Primary Completion 2026-05-05 (estimated)
Study Completion 2026-05-05 (estimated)
Updated on ClinicalTrials.gov 2025-12-22