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Clinical trial condition

GNE Myopathy

Explore clinical trials studying GNE Myopathy. Study availability and eligibility vary by location and protocol.

10 public trials 0 recruiting studies
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GNE Myopathy trial results

Completed

Efficacy Confirmation Study of NPC-09

NCT04671472

Conditions
Distal Myopathy With Rimmed Vacuoles (DMRV), Distal myopathy, Nonaka type, GNE Myopathy, +2 more
Locations
  • Nagoya University Hospital Nagoya, Aichi-ken, Japan
  • Tohoku University Hospital Sendai, Miyagi, Japan
  • Osaka University Hospital Suita, Osaka, Japan
  • National Center Hospital of Neurology and Psychiatry Hospita Kodaira, Tokyo, Japan
View Trial Details
Completed

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)

NCT02377921

Conditions
Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, Distal myopathy, Nonaka type, +2 more
Locations
  • University of California, Irvine Irvine, California, United States
  • Washington University School of Medicine St Louis, Missouri, United States
  • New York University School of Medicine New York, United States
  • Icahn School of Medicine at Mount Sinai New York, United States
View all 13 locations
View Trial Details
Completed

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)

NCT01784679

Conditions
Disease Attributes, Distal Myopathy With Rimmed Vacuoles (DMRV), Distal myopathy, Nonaka type, +7 more
Locations
  • University of California, Irvine Irvine, California, United States
  • Bulgarian Neuromuscular Disease Association Sofia, Bulgaria
  • McMaster University Hamilton, Ontario, Canada
  • Association Institut de Myologie Paris, France
View Trial Details
Completed

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

NCT01830972

Conditions
Distal myopathy, Nonaka type, GNE Myopathy, Hereditary Inclusion Body Myopathy (hIBM)
Locations
  • UCLA Medical Center Los Angeles, California, United States
  • Washington University School of Medicine St Louis, Missouri, United States
  • NYU Medical Center New York, United States
  • Hadassah University Hospital Jerusalem, Israel
View Trial Details
Completed

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

NCT01517880

Conditions
Distal myopathy, Nonaka type, GNE Myopathy, Hereditary Inclusion Body Myopathy
Locations
  • UCLA Medical Center Los Angeles, California, United States
  • Washington University School of Medicine St Louis, Missouri, United States
  • New York University School of Medicine New York, United States
  • Hadassah University Hospital Jerusalem, Israel
View Trial Details
Active, Not Recruiting

Multi-Center Study of ManNAc for GNE Myopathy

NCT04231266

Conditions
Distal myopathy, Nonaka type, GNE Myopathy
Locations
  • UCLA Los Angeles, California, United States
  • University of Iowa Iowa City, Iowa, United States
  • University of Kansas, Medical Center Kansas City, Kansas, United States
  • NIH Clinical Center Bethesda, Maryland, United States
View Trial Details

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