113. Muscular dystrophy Clinical trials / Disease details
Clinical trials : 622 / Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168
| 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 | NCT05004129 (ClinicalTrials.gov) | August 23, 2021 | 5/8/2021 | Safety and Efficacy of Tideglusib in Congenital Myotonic Dystrophy | A 52-Week, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital DM1 (REACH CDM X) | Congenital Myotonic Dystrophy | Drug: Tideglusib | AMO Pharma Limited | NULL | Enrolling by invitation | 6 Years | 17 Years | All | 56 | Phase 2/Phase 3 | United States;Australia;Canada;New Zealand |
| 2 | NCT03692312 (ClinicalTrials.gov) | March 3, 2021 | 16/3/2018 | Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy | A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM) | Congenital Myotonic Dystrophy | Drug: Tideglusib;Drug: Placebo | AMO Pharma Limited | NULL | Recruiting | 6 Years | 16 Years | All | 56 | Phase 2/Phase 3 | United States;Australia;Canada;New Zealand;United Kingdom |
| 3 | EUCTR2016-004623-23-GB (EUCTR) | 19/11/2018 | 08/02/2018 | A Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy | A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy - Efficacy & Safety of Tideglusib in Congenital Myotonic Dystrophy | Treatment of child and adolescent congenital myotonic dystrophy. MedDRA version: 20.0;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Tideglusib INN or Proposed INN: Tideglusib Other descriptive name: 4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione | AMO Pharma Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 56 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Canada;United Kingdom | ||
| 4 | NCT02858908 (ClinicalTrials.gov) | July 20, 2016 | 4/8/2016 | Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy | A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy | Myotonic Dystrophy 1 | Drug: Tideglusib | AMO Pharma Limited | NULL | Completed | 12 Years | 45 Years | All | 16 | Phase 2 | United Kingdom |
| 5 | EUCTR2016-000067-16-GB (EUCTR) | 20/04/2016 | 13/05/2016 | A clinical trial to investigate how safe and effective tideglusib is, as treatment for adolescents and adults with myotonic dystrophy diagnosed before they were 12 years old. | A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy | Treatment of adolescent and adult congenital and juvenile onset myotonic dystrophy MedDRA version: 19.1;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Tideglusib INN or Proposed INN: Tideglusib Other descriptive name: 4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione | AMO Pharma Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United Kingdom |