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NCT04248439 · ClinicalTrials.gov registry record · Phase 2
Gene Therapy for Fanconi Anemia, Complementation Group A
A Phase 2 study of Fanconi Anemia Complementation Group A, sponsored by Rocket Pharmaceuticals.
- Active
- Registry status
- Phase 2
- Development phase
- 5
- Enrollment target
- 2
- Study locations
NCT04248439: Active Phase 2 study of Fanconi Anemia Complementation Group A, sponsored by Rocket Pharmaceuticals.
NCT04248439 is a Phase 2 study of Fanconi Anemia Complementation Group A that is active but no longer recruiting, run by Rocket Pharmaceuticals. The registered enrollment target is 5 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (96% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04248439, a Phase 2 study of Fanconi Anemia Complementation Group A, is active but no longer recruiting, sponsored by Rocket Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 5 participants
- Enrollment target
- 2
- Study locations
Study 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.
Primary Outcome
Bone Marrow (BM) colony-forming cell (CFC) mitomycin-C (MMC) resistance ≥20% at 12 months post-infusion with a confirmatory result at 18 or 21 months (MMC at 10 nM concentration)
Conditions Studied
Interventions
- BIOLOGICAL RP-L102
Study Locations (2)
California
- Stanford University - Stanford
Minnesota
- University of Minnesota - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2020-07-15 |
| Est. Completion | 2026-05-05 |
| Phase | Phase 2 |
What the registry record for NCT04248439 still lists
NCT04248439 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (96% lower).
The record links to 1 condition, with Fanconi Anemia Complementation Group A appearing as the primary indexed condition, and to 1 intervention - of which RP-L102 is the first listed.
NCT04248439 reports a single indexed study location in California, Minnesota.
Frequently Asked Questions
What is clinical trial NCT04248439 about?
NCT04248439 is a clinical study titled "Gene Therapy for Fanconi Anemia, Complementation Group A". 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 lentiv...
What is the current status of trial NCT04248439?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 5 participants. The study started on 2020-07-15. Estimated completion is 2026-05-05.
What conditions does trial NCT04248439 study?
This clinical trial studies the following conditions: Fanconi Anemia Complementation Group A.
What interventions are being tested in trial NCT04248439?
The interventions under investigation include: RP-L102 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04248439?
This trial is sponsored by Rocket Pharmaceuticals, which has 10 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04248439 being conducted?
This trial has 2 study locations across California, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04248439's enrollment target sits among peer trials
5 2000th of 2000 the lowest of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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