Aceneuramic Acid Extended-Release Tablets

( DrugBank: - / KEGG DRUG: - )

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	8

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

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Showing 1 to 8 of 8 trials

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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2016-000360-42-BG (EUCTR)	26/04/2017	22/02/2017	A study to see if aceneuramic acidis safe and effective in treating people with Hereditary Inclusion ...	A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extend ...	GNE Myopathy, also known as Hereditary Inclusion Body Myopathy(HIBM), Distal Myopathy with Rimmed Va ...	Product Name: INN: aceneuramic acid; Common Name: Sialic Acid Product Code: UX001 INN or Proposed INN: ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	165	Phase 3	France;United States;Canada;Israel;Bulgaria;Italy;United Kingdom
2	EUCTR2016-000360-42-IT (EUCTR)	06/02/2017	07/02/2018	A study to see if aceneuramic acidis safe and effective in treating people with Hereditary Inclusion ...	A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extend ...	GNE Myopathy, also known as Hereditary Inclusion Body Myopatha\y (HIBM), Distal Myopathy with Rimmed ...	Product Name: Aceneuramic Acid; Common Name: Sialic Acid Product Code: UX001	ULTRAGENYX PHARMACEUTICAL INC.	NULL	Not Recruiting			Female: yes Male: yes	110	Phase 3	France;Canada;Israel;Bulgaria;United Kingdom;Italy
3	EUCTR2016-000360-42-GB (EUCTR)	02/08/2016	19/05/2016	A study to see if aceneuramic acidis safe and effective in treating people with Hereditary Inclusion ...	A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extend ...	GNE Myopathy, also known as Hereditary Inclusion Body Myopathy(HIBM), Distal Myopathy with Rimmed Va ...	Product Name: INN: aceneuramic acid; Common Name: Sialic Acid Product Code: UX001 INN or Proposed INN: ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	165	Phase 3	France;United States;Canada;Israel;Bulgaria;Italy;United Kingdom
4	EUCTR2014-005432-33-BG (EUCTR)	07/06/2016	20/04/2016	A study to see if sialic acidis safe and effective in treating people with Hereditary Inclusion Body ...	A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ...	GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed V ...	Product Name: Sialic acid(INN: aceneuramic acid) Product Code: UX001 INN or Proposed INN: ACENEURAMIC ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	89	Phase 3	France;United States;Canada;Israel;Bulgaria;Italy;United Kingdom
5	NCT02736188 (ClinicalTrials.gov)	May 2, 2016	4/4/2016	Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release(Ace-ER) Tabletsin Pat ...	Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended ...	Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type ...	Drug: Aceneuramic Acid Extended-Release Tablets	Ultragenyx Pharmaceutical Inc	NULL	Terminated	18 Years	N/A	All	143	Phase 3	United States;Bulgaria;Canada;France;Israel;Italy;United Kingdom
6	NCT02731690 (ClinicalTrials.gov)	April 29, 2016	24/3/2016	A Study to Evaluate the Safety of Aceneuramic AcidExtended Release (Ace-ER; UX001) Tabletsin Glucosa ...	A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic AcidExtended Release (Ace-ER) Table ...	Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type ...	Drug: Aceneuramic Acid Extended-Release	Ultragenyx Pharmaceutical Inc	NULL	Terminated	18 Years	N/A	All	42	Phase 2	United States;Bulgaria;Canada
7	EUCTR2014-005432-33-IT (EUCTR)	16/12/2015	19/01/2021	not available	A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ...	GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed V ...	Product Name: Acido Sialico - Sialic acid (INN: aceneuramic acid) Product Code: UX001	ULTRAGENYX PHARMACEUTICAL INC.	NULL	Not Recruiting			Female: yes Male: yes	89	Phase 3	France;United States;Canada;Bulgaria;Israel;United Kingdom;Italy
8	NCT02377921 (ClinicalTrials.gov)	May 20, 2015	27/2/2015	Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acidin Patients With G ...	A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ...	Hereditary Inclusion Body Myopathy;Distal Myopathy With Rimmed Vacuoles;Distal Myopathy, Nonaka Type ...	Drug: aceneuramic acid extended-release (Ace-ER);Drug: Placebo	Ultragenyx Pharmaceutical Inc	NULL	Completed	18 Years	55 Years	All	89	Phase 3	United States;Bulgaria;Canada;France;Israel;Italy;United Kingdom

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