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Through this multi-center study researchers will determine the optimal dose of the investigational drug 4D-310 for patients with Fabry disease, a rare inherited and progressive genetic disorder that leads to damage of the kidneys, heart, and brain. Fabry disease is caused by a change in the GLA gene, which provides cells with instructions to make an enzyme needed by the body. The 4D-310 drug contains an unchanged copy of the GLA gene which may help cells to create the needed enzyme.
Subject to certain exclusion criteria, this study is open to males ages 18 and older who have a confirmed diagnosis of classic Fabry disease.
Males: Ages 18 and up
Study participation will consist of periods for screening, treatment/observation, and long-term follow-up. Screening will determine eligibility and may take 45 days or longer. Once accepted into the study, participants will receive a dose of 4D-310 by intravenous infusion, requiring a 24-hour hospital stay for observation. After that, participants will need to return to the clinic every 1 to 2 weeks for the first 3 months and then less frequently for the balance of the study, which runs for 5 years.
Duration: 5 years or longer
4D Molecular Therapeutics
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Michele Graham, RN, Clinical Research Coordinator
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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