NCT04248439

Gene Therapy for Fanconi Anemia, Complementation Group A

A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

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.

Detailed description

This is a pediatric open-label Phase II clinical trial to assess the efficacy of a hematopoietic gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in subjects with FA-A. Enriched CD34+ hematopoietic stem cells will be transduced ex vivo with the therapeutic lentiviral vector and infused via intravenous infusion following transduction without any prior conditioning.

Conditions

• Fanconi Anemia Complementation Group A

Interventions

Type	Name	Description
BIOLOGICAL	RP-L102	CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a lentiviral vector carrying the FANCA gene

Timeline

• Start date: 2020-07-15
• Primary completion: 2026-05-05
• Completion: 2026-05-05
• First posted: 2020-01-30
• Last updated: 2025-12-22

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study