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

  
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PhaseCountries
1NCT04940572
(ClinicalTrials.gov)
September 1, 202111/5/2021Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram SyndromeAUDIOWOLF: 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 SyndromeDrug: DepakineCentre d'Etude des Cellules SouchesGenethonNot yet recruiting13 YearsN/AAll23Phase 2NULL
2EUCTR2017-001215-37-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 2France;Poland;United Kingdom
3NCT03717909
(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
4EUCTR2017-001215-37-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]Trade Name: Sodium Valproate Wockhardt 200mg Gastro-Resistant tablets
Product Name: Sodium Valproate Wockhardt 200mg Gastro-Resistant tablets
University of BirminghamNULLAuthorised-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): noFrance;Poland;Spain;United Kingdom
5NCT02829268
(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)Active, not recruiting5 Years60 YearsAll50Phase 1/Phase 2United States
6NCT02882477
(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
7NCT01302327
(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
8EUCTR2020-004594-43-FR
(EUCTR)
11/03/2021AUDIOWOLF: A phase II, open-label, efficacy study of daily administration of sodium valproate in patients clinically affected by Wolfram syndrome due to monogenic mutationAUDIOWOLF: 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-StemNULLNAFemale: yes
Male: yes
23Phase 2France
9EUCTR2017-001215-37-FR
(EUCTR)
03/02/2020Clinical 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 BirminghamNULLNAFemale: yes
Male: yes
70Phase 2France;Spain;Poland;United Kingdom