4 completed, 1 recruiting. 5 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 5 | Completed 4, Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Distal myopathy, Nonaka type: new trials by email Literature Review of Distal myopathy, Nonaka type trials Research Report on Distal myopathy, Nonaka type
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| First-in-human Study of UX016 in GNEM UX016, Placebo - GNE Myopathy | Phase 1 / Phase 2 | Recruiting | Ultragenyx Pharmaceutical Inc | 24 | 2026-10-01 | 2026-10-13 |
| Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) ManNAc - Hereditary Inclusion Body Myopathy (HIBM), GNE Myopathy | Phase 1 | Completed | National Human Genome Research Institute (NHGRI) | 26 | 2012-09-11 | 2017-10-19 |
| Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM) Sialic Acid Extended Release (SA-ER) Tablets, Sialic Acid Extended Release (SA-ER) Tables - Hereditary Inclusion Body Myopathy (HIBM) | Phase 1 | Completed | Ultragenyx Pharmaceutical Inc | 26 | 2011-07-01 | 2012-05-21 |
| Pharmacokinetic Study on N-acetylneuraminic Acid NPC-09 - Nonaka Myopathy, Hereditary Inclusion Body Myopathy | Phase 1 | Completed | Tohoku University | 6 | 2010-11-01 | 2011-09-08 |
| Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy Immune Globulin - Hereditary Inclusion Body Myopathy | Phase 1 | Completed | National Human Genome Research Institute (NHGRI) | 4 | 2005-09-16 | 2019-12-04 |
Immunoglobulins (1) · N-acetylmannosamine (1) · Tablets (1)
National Human Genome Research Institute (NHGRI) (2) · Ultragenyx Pharmaceutical Inc (2) · Tohoku University (1)
United States (4) · Japan (1)
Masashi Aoki (1) · Nuria Carrillo-Carrasco, M.D. (1) · William A Gahl, M.D. (1)