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Gene Therapy for Fanconi Anemia, Complementation Group A

StatusActive, Not Recruiting
PhasePhase 2
Started
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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.

Record History

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  1. Primary and study completion dates: May 2026 to May 5, 2026v4
  2. Primary completion date: Jun 2022 to May 2026v3 Show 6 more changesShow less
    • Primary endpoint revised
    • Trial status: Recruiting to Active, Not Recruiting
    • Study completion date: Jun 2023 to May 2026
    • Eligibility criteria shortened, about 36 words
    • Secondary endpoints: 3 to 10
    • Site details revised at 2 of 2 sites
  3. Primary completion date: Aug 2023 to Jun 2022
    • Start date changed from estimate of Feb 2020 to Jul 15, 2020
    • Study sites: 1 to 2
    • Study completion date: Aug 2023 to Jun 2023
    v2
  4. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT04248439
Lead Sponsor Rocket Pharmaceuticals Inc.
Conditions
(from sponsor)		 Fanconi Anemia Complementation Group A
Related terms Anemia, Bone Marrow Failure Disorders, Fanconi Syndrome
Enrollment 5 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov