30. Distal myopathy Clinical trials / Disease details
Clinical trials : 15 / Drugs : 17 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathway : 1
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02736188 (ClinicalTrials.gov) | May 2, 2016 | 4/4/2016 | 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) | Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) | Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type;GNE Myopathy;Quadriceps Sparing Myopathy | Drug: Aceneuramic Acid Extended-Release Tablets | Ultragenyx Pharmaceutical Inc | NULL | Terminated | 18 Years | N/A | All | 143 | Phase 3 | United States;Bulgaria;Canada;France;Israel;Italy;United Kingdom |