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

Location:

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