DDrare: Database of Drug Development for Rare Diseases

111. 先天性ミオパチー
［臨床試験数：7，薬物数：13（DrugBank：4），標的遺伝子数：1，標的パスウェイ数：8］

Searched query = "Congenital myopathy", "Nemaline myopathy", "Central core disease", "Minicore myopathy", "Myotubular myopathy", "Centronuclear myopathy", "Congenital fiber-type disproportion 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 = AT132; Acetylcysteine; DYN101; L-Tyrosine; N-acetylcysteine; RAAV8-Des-hMTM1; SALBUTAMOL SULFATE; Salbutamol; Sulfate; Tyrosine; Ventoline; Ventoline oral solution 0,4mg/ml; Ventoline tablet 2mg

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	NCT02362425 (ClinicalTrials.gov)	February 12, 2015	12/2/2015	Antioxidant Therapy in RYR1-Related Congenital Myopathy	Antioxidant Therapy in RYR1-Related Congenital Myopathy	Neuromuscular Disease	Drug: N-acetylcysteine;Drug: Placebo	National Institute of Nursing Research (NINR)	NULL	Completed	7 Years	N/A	All	63	Phase 1;Phase 2	United States

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

NCT02362425
(ClinicalTrials.gov)

February 12, 2015

12/2/2015

Antioxidant Therapy in RYR1-Related Congenital Myopathy

Neuromuscular Disease

Drug: N-acetylcysteine;Drug: Placebo

National Institute of Nursing Research (NINR)

NULL

Completed

7 Years

N/A

All

Phase 1;Phase 2

United States