Acido Sialico - 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1

30. 遠位型ミオパチー


臨床試験数 : 15 薬物数 : 17 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 1
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
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Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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PhaseCountries
1EUCTR2014-005432-33-IT
(EUCTR)
16/12/201519/01/2021not availableA 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) - not applicable 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: Acido Sialico - Sialic acid (INN: aceneuramic acid)
Product Code: UX001
ULTRAGENYX PHARMACEUTICAL INC.NULLNot RecruitingFemale: yes
Male: yes
89Phase 3France;United States;Canada;Bulgaria;Israel;United Kingdom;Italy