Testing effectiveness (Phase 2)Looking for participantsNCT07511556What this trial is testingFirst-in-human Study of UX016 in GNEMWho this might be right forGNE Myopathy Ultragenyx Pharmaceutical Inc 24
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
Early research (Phase 1)Study completedNCT01634750What this trial is testingPhase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)Who this might be right forHereditary Inclusion Body Myopathy (HIBM)GNE Myopathy National Human Genome Research Institute (NHGRI) 26
Large-scale testing (Phase 3)Study completedNCT01925209What this trial is testingEfficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM PatientsWho this might be right forSporadic Inclusion Body Myositis Novartis Pharmaceuticals 251
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