Aceneuramic Acid Extended-Release Tablets ( 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
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT02736188
(ClinicalTrials.gov)
May 2, 20164/4/2016Study 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)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)Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type;GNE Myopathy;Quadriceps Sparing MyopathyDrug: Aceneuramic Acid Extended-Release TabletsUltragenyx Pharmaceutical IncNULLTerminated18 YearsN/AAll143Phase 3United States;Bulgaria;Canada;France;Israel;Italy;United Kingdom