GNE Myopathy (DBCOND0055683)

Identifiers

Synonyms

Hereditary Inclusion Body Myopathy / Hereditary Inclusion Body Myopathy (HIBM) / Distal Myopathy, Nonaka Type / Nonaka Myopathy / Nonaka Disease / GNE myopathy / Inclusion body myopathy 2 (disorder)

Associated Data

Indicated Drugs and Targets

Not Available

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT02731690	A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment	Aceneuramic acid	treatment	2	terminated
NCT02377921	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)	Aceneuramic acid	treatment	3	completed
NCT01784679	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)	No drug interventions	Not Available	Not Available	completed
NCT02736188	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)	Aceneuramic acid	treatment	3	terminated
NCT04009226	International GNE Myopathy Patient Registry	No drug interventions	Not Available	Not Available	unknown_status
NCT01236898	Pharmacokinetic Study on N-acetylneuraminic Acid	Aceneuramic acid	treatment	1	completed
NCT00195637	Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy	No drug interventions	treatment	1	completed
NCT01517880	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	No drug interventions	treatment	2	completed
NCT01359319	Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)	No drug interventions	treatment	1	completed
NCT04671472	Efficacy Confirmation Study of NPC-09	Aceneuramic acid	treatment	3	completed
NCT01634750	Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)	N-acetylmannosamine	treatment	1	completed
NCT01830972	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	No drug interventions	treatment	2	completed
NCT04231266	Multi-Center Study of ManNAc for GNE Myopathy	N-acetylmannosamine	treatment	2	active_not_recruiting
NCT02346461	An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy	N-acetylmannosamine	treatment	2	completed
NCT01417533	A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases	No drug interventions	Not Available	Not Available	recruiting