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 233. ウォルフラム症候群 [臨床試験数:4,薬物数:10(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 of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03717909December 28, 201821 January 2019Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram SyndromeA Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram SyndromeWolfram SyndromeDrug: Sodium Valproate 200Mg E/C Tablet;Drug: Sodium Valproate matched placeboUniversity of BirminghamRecruiting5 YearsN/AAll70Phase 2United Kingdom
2NCT02829268January 201727 May 2019A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram SyndromeA Phase 1b Safety Trial of Dantronele Sodium in Pediatric and Adult Patients With Wolfram SyndromeWolfram Syndrome;Diabetes Mellitus;Optic Nerve Atrophy;AtaxiaDrug: dantrolene sodiumWashington University School of MedicineNational Institutes of Health (NIH)Recruiting5 Years60 YearsAll50Phase 1/Phase 2United States
3NCT02882477December 20165 September 2016Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based TherapyTreatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based TherapyDiabetes Mellitus;Iron Metabolism Disorders;Gastroduodenal Ulcer;Optic Atrophy;Sensorineural Hearing Loss;Platelet DysfunctionDrug: Deferiprone;Drug: Acetylcysteine;Drug: Sitagliptin and MetforminHadassah Medical OrganizationNot recruiting3 YearsN/ABoth20Phase 2/Phase 3Israel
4NCT01302327March 201119 February 2015GLP Analogs for Diabetes in Wolfram Syndrome PatientsDiabetes Mellitus Associated With Genetic Syndrome;Wolfram SyndromeDrug: ExenatideHadassah Medical OrganizationNot recruiting18 YearsN/ABoth10N/A