233. ウォルフラム症候群 Wolfram syndrome Clinical trials / Disease details
臨床試験数 : 11 / 薬物数 : 17 - (DrugBank : 8) / 標的遺伝子数 : 12 - 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 | NCT05676034 (ClinicalTrials.gov) | March 2023 | 22/12/2022 | AMX0035 in Adult Patients With Wolfram Syndrome | A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome | Wolfram Syndrome | Drug: AMX0035 | Amylyx Pharmaceuticals Inc. | NULL | Recruiting | 17 Years | N/A | All | 12 | Phase 2 | United States |
| 2 | NCT05659368 (ClinicalTrials.gov) | January 2023 | 5/12/2022 | Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1 | Towards a Personalized Precision Medicine in Rare Disease: Tirzepatide (a Dual Glucose-dependent Insulinotropic Polypeptide and Glucagon-like Peptide-1 Receptor Agonist) Monotherapy in Patients With Wolfram Syndrome Type 1 | Wolfram Syndrome | Drug: Tirzepatide | Ospedale San Raffaele | NULL | Not yet recruiting | 5 Years | N/A | All | 10 | Phase 2 | NULL |
| 3 | NCT04940572 (ClinicalTrials.gov) | November 26, 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 | Recruiting | 13 Years | N/A | All | 23 | Phase 2 | France |
| 4 | 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 | ||
| 5 | 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 |
| 6 | 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 | ||
| 7 | 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) | Active, not recruiting | 5 Years | 60 Years | All | 50 | Phase 1/Phase 2 | United States |
| 8 | 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 |
| 9 | 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 | |
| 10 | 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 | |||
| 11 | 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 |