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 | EUCTR2014-005432-33-IT (EUCTR) | 16/12/2015 | 19/01/2021 | not available | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) - not applicable | GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka's disease, or quadriceps sparing myopathy (QSM) MedDRA version: 20.0;Level: LLT;Classification code 10075048;Term: Hereditary inclusion body myopathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Acido Sialico - Sialic acid (INN: aceneuramic acid) Product Code: UX001 | ULTRAGENYX PHARMACEUTICAL INC. | NULL | Not Recruiting | Female: yes Male: yes | 89 | Phase 3 | France;United States;Canada;Bulgaria;Israel;United Kingdom;Italy |