DDrare: Database of Drug Development for Rare Diseases

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.

Drug = ACENEURAMIC ACID; Aceneuramic Acid; Aceneuramic Acid Extended-Release; Aceneuramic Acid Extended-Release Tablets; Aceneuramic acid; Aceneuramic acid extended-release (Ace-ER); Common Name: Sialic Acid; Deflazacort; INN: aceneuramic acid; N-acetylneuraminic acid; NPC-09; SA; Sialic acid; Sialic acid (INN: aceneuramic acid); Sialic acid, N-acetylneuraminic acid; UX001

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	NCT02736188 (ClinicalTrials.gov)	May 2, 2016	4/4/2016	Study 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 Myopathy	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

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

NCT02736188
(ClinicalTrials.gov)

May 2, 2016

4/4/2016

Study 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 Myopathy

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