Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

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

9 trials

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 9 of 9 results

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
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
Testing effectiveness (Phase 2)Looking for participantsNCT07511556
What this trial is testing

First-in-human Study of UX016 in GNEM

Who this might be right for
GNE Myopathy
Ultragenyx Pharmaceutical Inc 24
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)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
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)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)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
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