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	NCT03717909 (ClinicalTrials.gov)	December 28, 2018	11/10/2018	Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome	A Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome	Wolfram Syndrome	Drug: Sodium Valproate 200Mg E/C Tablet;Drug: Sodium Valproate matched placebo	University of Birmingham	NULL	Recruiting	5 Years	N/A	All	70	Phase 2	France;Poland;Spain;United Kingdom

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NCT03717909
(ClinicalTrials.gov)

December 28, 2018

11/10/2018

Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome

A Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome

Wolfram Syndrome

Drug: Sodium Valproate 200Mg E/C Tablet;Drug: Sodium Valproate matched placebo

University of Birmingham

NULL

Recruiting

5 Years

N/A

All

Phase 2

France;Poland;Spain;United Kingdom