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Sialic acid (inn: aceneuramic acid)    (DrugBank: Aceneuramic acid)

1 disease
告示番号疾患名（ページ内リンク）臨床試験数
30遠位型ミオパチー2

30. 遠位型ミオパチー　［臨床試験数：13，薬物数：16（DrugBank：3），標的遺伝子数：1，標的パスウェイ数：1］
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.
2 / 13 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2015-004553-41-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
2EUCTR2014-005432-33-BG
(EUCTR)		07/06/201620/04/2016A study to see if sialic acid is safe and effective in treating people with Hereditary Inclusion Body Myopathy (HIBM), a rare muscle disease.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) 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: Sialic acid (INN: aceneuramic acid)
Product Code: UX001
INN or Proposed INN: ACENEURAMIC ACID		Ultragenyx Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		89Phase 3France;United States;Canada;Israel;Bulgaria;Italy;United Kingdom