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.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03717909 | December 28, 2018 | 21 January 2019 | 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 | Recruiting | 5 Years | N/A | All | 70 | Phase 2 | United Kingdom | |
| 2 | NCT02829268 | January 2017 | 27 May 2019 | A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome | A Phase 1b Safety Trial of Dantronele 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) | Recruiting | 5 Years | 60 Years | All | 50 | Phase 1/Phase 2 | United States |
| 3 | NCT02882477 | December 2016 | 5 September 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 | Not recruiting | 3 Years | N/A | Both | 20 | Phase 2/Phase 3 | Israel | |
| 4 | NCT01302327 | March 2011 | 19 February 2015 | GLP Analogs for Diabetes in Wolfram Syndrome Patients | Diabetes Mellitus Associated With Genetic Syndrome;Wolfram Syndrome | Drug: Exenatide | Hadassah Medical Organization | Not recruiting | 18 Years | N/A | Both | 10 | N/A |