NIH Will Test Antioxidant Drug in Central Core Disease

A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old

Article Highlights:
  • Antioxidants, which interfere with potentially dangerous oxidation reactions in cells, could be helpful in central core disease (CCD), a disorder involving weakness due to defects in the ryanodine receptor, a calcium release channel inside cells.
  • The antioxidant being tested has FDA approval for other indications.
  • The trial is being conducted only at NIH in Bethesda, Md.
by Margaret Wahl on February 13, 2015 - 11:50am

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: 

  • be at least 7 years old
  • be able to walk
  • have a diagnosis of CCD
  • have had a muscle biopsy confirming CCD

Participants in the antioxidant trial must not:

  • have a history of liver or lung disease, ulcers, trouble swallowing or problems in the esophagus
  • be pregnant or plan to become pregnant
  • be nursing or breastfeeding

To participate

For more information, contact Irene Arveson at NIH at irene.arveson@nih.gov or (301) 451-4881.

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