30. Distal myopathy
13 clinical trials,   16 drugs   (DrugBank: 3 drugs),   1 drug target gene,   1 drug target pathway

Searched query = "Distal myopathy", "Distal muscular dystrophy", "Miyoshi myopathy", "Distal dysferlinopathy", "DMRV/GNE myopathy", "Oculopharyngodistal myopathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
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PhaseCountries
1EUCTR2016-000360-42-BG
(EUCTR)
26/04/201722/02/2017A study to see if aceneuramic acid is safe and effective in treating people with Hereditary Inclusion Body Myopathy (HIBM), a rare muscle disease.A 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) GNE Myopathy, also known as Hereditary Inclusion Body Myopathy(HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka'sdisease, or quadriceps sparing myopathy (QSM)
MedDRA version: 19.1;Level: LLT;Classification code 10075048;Term: Hereditary inclusion body myopathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.1;Level: PT;Classification code 10077945;Term: GNE myopathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: INN: aceneuramic acid; Common Name: Sialic Acid
Product Code: UX001
INN or Proposed INN: ACENEURAMIC ACID
Other descriptive name: sialic acid, N-acetylneuraminic acid
Ultragenyx Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
165Phase 3France;United States;Canada;Israel;Bulgaria;Italy;United Kingdom
2JPRN-UMIN000026354
2017/03/2820/03/2017A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM)A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) - A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) GNE myopathy (Distal myopathy with rimmed vacuoles (DMRV), hereditary inclusion body myopathy (hIBM) or Nonaka disease)SA-ER 2g 3 times oral dosing a day for 48 consecutive weeksTohoku University Hospital, Department of NeurologyNobelpharma Co., Ltd.Complete: follow-up complete20years-old50years-oldMale and Female20Phase 2;Phase 3Japan
3EUCTR2015-004553-41-BG
(EUCTR)
05/01/201714/09/2016A study to see if Aceneuramic Acid is safe in treating people with GNE Myopathy (GNEM) patients (a rare muscle disease also known as Hereditary Inclusion Body Myopathy (HIBM)) with severe ambulatory impairment.A Phase 2 Open-label study to Evaluate the Safety of Aceneuramic Acid ExtendedRelease (Ace-ER) Tablets in GNE Myopathy (GNEM) (also known as HereditaryInclusion Body Myopathy (HIBM)) patients with Severe Ambulatory Impairment 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: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: PT;Classification code 10077945;Term: GNE myopathy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Sialic acid (INN: aceneuramic acid)
Product Code: UX001
INN or Proposed INN: ACENEURAMIC ACID
Other descriptive name: sialic acid; N-acetylneuraminic acid
Ultragenyx Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
45Phase 2United States;Canada;Bulgaria
4JPRN-UMIN000020683
2016/02/0125/01/2016A Phase II/III Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) GNE myopathy (Distal myopathy with rimmed vacuoles (DMRV), hereditary inclusion body myopathy (hIBM) or Nonaka disease)SA-ER 2g 3 times oral dosing a day for 48 consecutive weeks
placebo 3 times oral dosing a day for 48 consecutive weeks
Tohoku University Hospital, Department of NeurologyNobelpharma Co., Ltd.Complete: follow-up complete20years-old50years-oldMale and Female20Phase 2;Phase 3Japan
5NCT01236898
(ClinicalTrials.gov)
November 20108/11/2010Pharmacokinetic Study on N-acetylneuraminic AcidPharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)Nonaka Myopathy;Hereditary Inclusion Body MyopathyDrug: NPC-09Tohoku UniversityNULLCompleted20 Years40 YearsBoth6Phase 1Japan