Investigators at the National Institute of Neurological Disorders and Stroke (NINDS) are conducting a research study to evaluate the safety and effectiveness of an experimental drug, called OGT 918, and enzyme replacement therapy (ERT) compared to ERT alone in the treatment of Gaucher disease type III. Scientists hope OGT 918 will improve the neurological symptoms of the disease.
Eligible participants must be over 4 years of age and able to swallow capsules, and must have been receiving stable ERT for at least 6 months. Persons who are under 4 years of age, have type I or type II Gaucher disease, or are pregnant or breastfeeding are not eligible.
The study will take place at the National Institutes of Health (NIH) Clinical Center in Bethesda, MD. All study-related expenses will be paid by the NIH. This study is carried out in compliance with safety and testing standards of the U.S. Department of Health and Human Services.
For more information, contact Dr. Raphael Schiffmann or Val Robinson, R.N., NINDS, NIH, Building 10, Room 3D03, 10 Center Drive MSC 1260, Bethesda, MD 20892-1260, telephone: 301-496-1465 or 1-800-258-0299 (toll-free); fax: 301-480-8354.