HomeTrialNCT04248439
🔬NCT04248439Phase 2ACTIVE NOT RECRUITING

Gene Therapy for Fanconi Anemia, Complementation Group A

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.

Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment5 participants
Primary Completion
Lead SponsorRocket Pharmaceuticals Inc.
Data Retrieved2026-04-11 04:25:37.504939+00:00
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