Joseph M. Sanzari Children’s Hospital One of Two Nationwide Sites for Fabry Disease Study

Study Evaluating New One-Time Gene Therapy Approach for Fabry Disease

Hackensack Meridian Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center was selected as one of the two current sites in the nation to be a part of a pioneering study of a new one-time therapy for Fabry disease. The study is titled “An Open-Label, Multinational Study of the Efficacy and Safety of Ex Vivo, Lentiviral Vector-Mediated Gene Therapy Avr-Rd-01 for Treatment-Naïve Subjects With Classic Fabry Disease.”

In individuals diagnosed with Fabry disease, fatty molecules, known as globotriaosylceramide (Gb3 or GL-3), accumulate inside the patient’s cells, due to a lack of the enzyme alpha-galactosidase A. If untreated, this can lead to kidney failure.

The goal of the study is to provide a one-time therapy for the condition versus the current cadence of intravenous enzyme replacement therapy every other week. The trial treatment involves introducing a functional gene (GLA gene) to the patient and for the gene to continue to function and produce the desired protein (enzyme), alpha-galactosidase A.

Learn More about pediatric nephrology advances at Hackensack Meridian Children’s Health.