Fabry Disease Treatment Using 4D-310 Gene Therapy – Phase I/II

Trial of Gene Therapy 4D-310 in Adult Males with Classic Fabry Disease

Protocol Description

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.

Study Level: Phase I/II

Eligibility Criteria

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.
Visits: 24
Duration: 5 years or longer

Status: Open to Enrollment

Source(s) of Support

4D Molecular Therapeutics

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Michele Graham, RN, Clinical Research Coordinator