233. ウォルフラム症候群 Wolfram syndrome Clinical trials / Disease details
臨床試験数 : 9 / 薬物数 : 15 - (DrugBank : 7) / 標的遺伝子数 : 11 - 標的パスウェイ数 : 41
| 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 | NCT04940572 (ClinicalTrials.gov) | September 1, 2021 | 11/5/2021 | Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome | AUDIOWOLF: A Phase II, Open-label, Efficacy Study of Daily Administration of Sodium Valproate in Patients Clinically Affected by Wolfram Syndrome Due to Monogenic Mutation. | Wolfram Syndrome | Drug: Depakine | Centre d'Etude des Cellules Souches | Genethon | Not yet recruiting | 13 Years | N/A | All | 23 | Phase 2 | NULL |
| 2 | 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 | France;Poland;United Kingdom | ||
| 3 | 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 |
| 4 | 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] | Trade Name: Sodium Valproate Wockhardt 200mg Gastro-Resistant tablets Product Name: Sodium Valproate Wockhardt 200mg Gastro-Resistant tablets | University of Birmingham | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Poland;Spain;United Kingdom | ||
| 5 | EUCTR2020-004594-43-FR (EUCTR) | 11/03/2021 | AUDIOWOLF: A phase II, open-label, efficacy study of daily administration of sodium valproate in patients clinically affected by Wolfram syndrome due to monogenic mutation | AUDIOWOLF: A phase II, open-label, efficacy study of daily administration of sodium valproate in patients clinically affected by Wolfram syndrome due to monogenic mutation - AUDIOWOLF | Wolfram syndrome MedDRA version: 20.0;Level: PT;Classification code 10078338;Term: Wolfram syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: DEPAKINE CHRONO® 500 mg Product Name: DEPAKINE CHRONO 500 mg | CECS/I-Stem | NULL | NA | Female: yes Male: yes | 23 | Phase 2 | France | |||
| 6 | EUCTR2017-001215-37-FR (EUCTR) | 03/02/2020 | 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 Wockhardt 200mg Gastro-Resistant tablets Product Name: Sodium Valproate Wockhardt 200mg Gastro-Resistant tablets | University of Birmingham | NULL | NA | Female: yes Male: yes | 70 | Phase 2 | France;Spain;Poland;United Kingdom |