National Institutes of Health: Muscular Diseases trials

The registry lists 14 Muscular Diseases studies with National Institutes of Health as lead sponsor. 3 are open to participants now and 2 have results posted.

Phase 1 4Phase 2 4Phase 3 1No phase 8(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• A Natural History Study of RYR1-Related DisordersRecruitingNCT06287762
• Data Collection of Standard Care of Patients in the EMG SectionRecruitingNCT05041387
• Study of Inherited Neurological DisordersRecruitingNCT00004568
• Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult DermatomyositisCompletedNCT03267277Phase 2/3Results posted
• Antioxidant Therapy in RYR1-Related Congenital MyopathyCompletedNCT02362425Phase 1/2Results posted
• Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of CystinosisWithdrawnNCT02124070Phase 1/2
• Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)CompletedNCT01634750Phase 1
• Investigations Into ISCU Myopathy or Iron Sulfur Scaffold U Protein MyopathyCompletedNCT01547767
• Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular DystrophyCompletedNCT01451281
• The Effects of Exercise Versus Inactivity on People With Mitochondrial Muscle DiseaseStatus unknownNCT00457314Phase 2
• Intravenous Immune Globulin to Treat Hereditary Inclusion Body MyopathyCompletedNCT00195637Phase 1
• Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial MyopathyCompletedNCT00004770Not applicable
• Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic AcidemiaCompletedNCT00004353
• Genetic and Family Studies of Inherited Muscle DiseasesCompletedNCT00001331