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.
| 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 | EUCTR2017-001215-37-PL (EUCTR) | 13/02/2020 | 10/10/2019 | 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] | Trade Name: Sodium valproate 200 mg gastro-resistant tablets | University of Birmingham | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 2 | Poland;United Kingdom | ||
| 2 | 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 |
| 3 | EUCTR2017-001215-37-GB (EUCTR) | 19/03/2018 | 21/06/2018 | 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 Birmingham | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 2 | France;Poland;Spain;United Kingdom | |||
| 4 | NCT02829268 (ClinicalTrials.gov) | January 2017 | 6/7/2016 | A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome | A Phase 1b/2a Trial of Dantrolene 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);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Recruiting | 5 Years | 60 Years | All | 50 | Phase 1;Phase 2 | United States |
| 5 | 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 |
| 6 | NCT01302327 (ClinicalTrials.gov) | March 2011 | 22/2/2011 | GLP Analogs for Diabetes in Wolfram Syndrome Patients | Diabetes Mellitus Associated With Genetic Syndrome;Wolfram Syndrome | Drug: Exenatide | Hadassah Medical Organization | NULL | Not yet recruiting | 18 Years | N/A | Both | 10 | N/A | NULL |