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	NCT02882477 (ClinicalTrials.gov)	December 2016	17/7/2016	Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy	Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy	Diabetes Mellitus;Iron Metabolism Disorders;Gastroduodenal Ulcer;Optic Atrophy;Sensorineural Hearing Loss;Platelet Dysfunction	Drug: Deferiprone;Drug: Acetylcysteine;Drug: Sitagliptin and Metformin	Hadassah Medical Organization	NULL	Not yet recruiting	3 Years	N/A	Both	20	Phase 2;Phase 3	Israel

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

NCT02882477
(ClinicalTrials.gov)

December 2016

17/7/2016

Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy

Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy

Diabetes Mellitus;Iron Metabolism Disorders;Gastroduodenal Ulcer;Optic Atrophy;Sensorineural Hearing Loss;Platelet Dysfunction

Drug: Deferiprone;Drug: Acetylcysteine;Drug: Sitagliptin and Metformin

Hadassah Medical Organization

NULL

Not yet recruiting

3 Years

N/A

Both

Phase 2;Phase 3

Israel