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Results for “Distal Myopathy With Rimmed Vacuoles (DMRV)”

8 trials

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Showing 8 of 8 results

Testing effectiveness (Phase 2)Ended earlyNCT02731690
What this trial is testing

Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Who this might be right for
Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka Type+3 more
Ultragenyx Pharmaceutical Inc 42
Not applicableStudy completedNCT01784679
What this trial is testing

GNE-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 for
Hereditary Inclusion Body MyopathyGNE MyopathyNonaka Disease+2 more
Ultragenyx Pharmaceutical Inc 319
Large-scale testing (Phase 3)Ended earlyNCT02736188
What this trial is testing

Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Who this might be right for
Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka Type+2 more
Ultragenyx Pharmaceutical Inc 143
Testing effectiveness (Phase 2)Active Not RecruitingNCT04231266
What this trial is testing

Multi-Center Study of ManNAc for GNE Myopathy

Who this might be right for
GNE Myopathy
Leadiant Biosciences, Inc. 54
Large-scale testing (Phase 3)Study completedNCT02377921
What this trial is testing

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Who this might be right for
Hereditary Inclusion Body MyopathyDistal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka Type+1 more
Ultragenyx Pharmaceutical Inc 89
Testing effectiveness (Phase 2)Study completedNCT01517880
What this trial is testing

Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Who this might be right for
GNE MyopathyHereditary Inclusion Body Myopathy
Ultragenyx Pharmaceutical Inc 46
Large-scale testing (Phase 3)Study completedNCT04671472
What this trial is testing

Efficacy Confirmation Study of NPC-09

Who this might be right for
GNE MyopathyDistal Myopathy With Rimmed Vacuoles (DMRV)Hereditary Inclusion Body Myopathy (hIBM)+1 more
Nobelpharma 14
Early research (Phase 1)Study completedNCT01236898
What this trial is testing

Pharmacokinetic Study on N-acetylneuraminic Acid

Who this might be right for
Nonaka MyopathyHereditary Inclusion Body Myopathy
Tohoku University 6