Not applicableStudy completedNCT04009226What this trial is testingInternational GNE Myopathy Patient RegistryWho this might be right forGNE MyopathyHereditary Inclusion Body Myopathy Newcastle University 430
Not applicableStudy completedNCT01902940What this trial is testingNatural History in CCFDN and IBM SyndromesWho this might be right forInclusion Body Myositis, SporadicInclusion Body Myopathy, Autosomal-recessiveInclusion Body Myopathy, Autosomal-dominant+1 more Ludwig-Maximilians - University of Munich 350
Testing effectiveness (Phase 2)Study completedNCT01517880What this trial is testingEvaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body MyopathyWho this might be right forGNE MyopathyHereditary Inclusion Body Myopathy Ultragenyx Pharmaceutical Inc 46
Not applicableStudy completedNCT01784679What this trial is testingGNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)Who this might be right forHereditary Inclusion Body MyopathyGNE MyopathyNonaka Disease+2 more Ultragenyx Pharmaceutical Inc 319
Not applicableStudy completedNCT01417533What this trial is testingA Natural History Study of Patients With GNE Myopathy and GNE-Related DiseasesWho this might be right forGNE MyopathyGNE Related Diseases National Human Genome Research Institute (NHGRI) 78
Large-scale testing (Phase 3)Study completedNCT04671472What this trial is testingEfficacy Confirmation Study of NPC-09Who this might be right forGNE MyopathyDistal Myopathy With Rimmed Vacuoles (DMRV)Hereditary Inclusion Body Myopathy (hIBM)+1 more Nobelpharma 14