GNE Myopathy

13 registered clinical trials studyying GNE Myopathy — 1 currently recruiting.

Status	Trial	Sponsor	Phase
Not Yet Recruiting	First-in-human Study of UX016 in GNEM NCT07511556	Ultragenyx Pharmaceutical Inc	Phase 1 / Phase 2
Active Not Recruiting	Multi-Center Study of ManNAc for GNE Myopathy NCT04231266	Leadiant Biosciences, Inc.	Phase 2
Completed	Efficacy Confirmation Study of NPC-09 NCT04671472	Nobelpharma	Phase 3
Terminated	Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients Wi NCT02736188	Ultragenyx Pharmaceutical Inc	Phase 3
Terminated	A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UD NCT02731690	Ultragenyx Pharmaceutical Inc	Phase 2
Completed	Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamin NCT02377921	Ultragenyx Pharmaceutical Inc	Phase 3
Completed	An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy NCT02346461	National Human Genome Research Institute (NHGRI)	Phase 2
Unknown	International GNE Myopathy Patient Registry NCT04009226	Newcastle University	—
Completed	An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic A NCT01830972	Ultragenyx Pharmaceutical Inc	Phase 2
Completed	GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History S NCT01784679	Ultragenyx Pharmaceutical Inc	—
Completed	Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) NCT01634750	National Human Genome Research Institute (NHGRI)	Phase 1
Completed	A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tabl NCT01517880	Ultragenyx Pharmaceutical Inc	Phase 2
Completed	A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases NCT01417533	National Human Genome Research Institute (NHGRI)	—