A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old
The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as ryanodine receptor 1 (RYR1). The disease is associated with weakness and susceptibility to dangerous reactions to anesthesia ("malignant hyperthermia").
The antioxidant study, which is being conducted in Bethesda, Md., involves three outpatient visits of four to six days each to the NIH campus over the course of a year. Each participant will be assigned to receive either the study drug or a placebo three times a day for six months. Costs for all study-related tests, as well as travel and accommodations, are covered by the study. Antioxidants interfere with a chemical reaction called "oxidation," which can damage cells.
Participants in the antioxidant trial must:
Participants in the antioxidant trial must not:
For more information, contact Irene Arveson at NIH at firstname.lastname@example.org or (301) 451-4881.