Gene Therapy for Fanconi Anemia, Complementation Group A

Trial ID or NCT#

NCT04248439

Status

not recruiting iconNOT RECRUITING

Purpose

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.

Official Title

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

Eligibility Criteria

Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
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Investigator(s)

Rajni Agarwal
Professor of Pediatrics (Stem Cell Transplantation)
Agnieszka Czechowicz, MD, PhD

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Contact

Elisabeth Merkel

View on ClinicalTrials.gov