233. Wolfram syndrome
6 clinical trials,   12 drugs   (DrugBank: 7 drugs),   11 drug target genes,   40 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-001215-37-PL
(EUCTR)
13/02/202010/10/2019Clinical Trial of a Treatment to Slow Disease Progression Compared to Usual Standard of Care in Children and Adults with WolframA pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome - Treat Wolfram Wolfram syndrome;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Trade Name: Sodium valproate 200 mg gastro-resistant tabletsUniversity of BirminghamNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
70Phase 2Poland;United Kingdom
2NCT03717909
(ClinicalTrials.gov)
December 28, 201811/10/2018Efficacy 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 BirminghamNULLRecruiting5 YearsN/AAll70Phase 2France;Poland;Spain;United Kingdom
3EUCTR2017-001215-37-GB
(EUCTR)
19/03/201821/06/2018 Clinical Trial of a Treatment to Slow Disease Progression Compared to Usual Standard of Care in Children and Adults with Wolfram.A pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome - Treat Wolfram Wolfram syndrome;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]University of BirminghamNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
70Phase 2France;Poland;Spain;United Kingdom
4NCT02829268
(ClinicalTrials.gov)
January 20176/7/2016A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram SyndromeA Phase 1b/2a Trial of Dantrolene 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);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Recruiting5 Years60 YearsAll50Phase 1;Phase 2United States
5NCT02882477
(ClinicalTrials.gov)
December 201617/7/2016Treatment 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 OrganizationNULLNot yet recruiting3 YearsN/ABoth20Phase 2;Phase 3Israel
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT01302327
(ClinicalTrials.gov)
March 201122/2/2011GLP Analogs for Diabetes in Wolfram Syndrome PatientsDiabetes Mellitus Associated With Genetic Syndrome;Wolfram SyndromeDrug: ExenatideHadassah Medical OrganizationNULLNot yet recruiting18 YearsN/ABoth10N/ANULL