HomeTrialNCT04069533
🔬NCT04069533Phase 2ACTIVE NOT RECRUITING

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

Study Summary

This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric 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.

Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment7 participants
Primary Completion
Lead SponsorRocket Pharmaceuticals Inc.
Data Retrieved2026-04-11 04:25:37.504931+00:00
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