DDrare: Database of Drug Development for Rare Diseases

233. ウォルフラム症候群
［臨床試験数：6，薬物数：12（DrugBank：7），標的遺伝子数：11，標的パスウェイ数：40］

Searched query = "Wolfram syndrome", "Diabetes Insipidus, Diabetes mellitus, optic atrophy, and deafness syndrome", "DIDMOAD syndrome"
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 = Acetylcysteine; Dantrolene; Dantrolene sodium; Deferiprone; Exenatide; Metformin; Sitagliptin; Sitagliptin and Metformin; Sodium Valproate 200Mg E/C Tablet; Sodium Valproate Wockhardt 200mg Gastro-Resistant tablets; Sodium valproate 200 mg gastro-resistant tablets; Valproate

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	NCT02829268 (ClinicalTrials.gov)	January 2017	6/7/2016	A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome	A Phase 1b/2a Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome	Wolfram Syndrome;Diabetes Mellitus;Optic Nerve Atrophy;Ataxia	Drug: dantrolene sodium	Washington University School of Medicine	National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Recruiting	5 Years	60 Years	All	50	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

NCT02829268
(ClinicalTrials.gov)

January 2017

6/7/2016

A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

A Phase 1b/2a Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

Wolfram Syndrome;Diabetes Mellitus;Optic Nerve Atrophy;Ataxia

Drug: dantrolene sodium

Washington University School of Medicine

National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Recruiting

5 Years

60 Years

All

Phase 1;Phase 2

United States