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Ryanodine Receptor-1 (RYR1)Related Myopathy Research Study, 15-NR-0072 Volunteers Needed

The National Institute of Nursing Research (NINR) at the National Institutes of Health (NIH) in Bethesda, Maryland, is conducting a research study to determine if a drug that has been approved by the FDA for other indications reduces the severity of some symptoms in people with ryanodine receptor 1-related myopathy (RYR1-RM).

You may be eligible if you:

  • Are 7 years of age or older (Parents must give permission for minors to participate)
  • Are able to walk
  • Have a genetic diagnosis of RYR1-related myopathy, including congenital myopathy , or if anyone of your family does
  • Have had a muscle biopsy confirming RYR1-related myopathy, or if anyone in your family does

You may not be eligible if you:

  • Have a history of liver or lung disease
  • Have a history of ulcers, trouble swallowing, or have problems with your esophagus
  • Are pregnant or are planning to become pregnant
  • Are nursing or breastfeeding

The study involves:

  • Three outpatient visits to the NIH campus over one year
  • After your initial visit, you will be seen twice, 6 months apart
  • Each visit will take 4-6 days
  • You will be required to take the drug or placebo three times a day for 6 months
  • Travel and accommodation expenses will be covered for participants
  • All study-related tests are provided at no cost


The NIH Clinical Center, America’s research hospital, is located on the Metro red line (Medical Center station) in Bethesda, Maryland.

For more information contact:

Irene Arveson, BSN, RN
E-mail: [email protected]
Phone #: 301-451-4881
Online: clinicaltrials.gov
Refer to study 15-NR-0072

This item filed in the following categories:
  • General
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