113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 622 / 薬物数 : 485 - (DrugBank : 99) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 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 | NCT03603171 (ClinicalTrials.gov) | July 1, 2018 | 17/7/2018 | Clinical Outcome Measures in Myotonic Dystrophy Type 2 | Observational Trial in Myotonic Dystrophy Type 2 to Define Specific Clinical Outcome Measures | Myotonic Dystrophy Type 2 | Diagnostic Test: DM1-ActivC;Diagnostic Test: R-PAct;Diagnostic Test: Beck depression inventory;Diagnostic Test: McGill pain questionnaire;Diagnostic Test: Brief Pain Inventory Short-Form;Diagnostic Test: Fatigue and Daytime Sleepiness Scale;Diagnostic Test: Myotonia Behaviour scale;Diagnostic Test: Hand opening time;Diagnostic Test: Pressure pain threshold;Diagnostic Test: Manual muscle testing;Diagnostic Test: Quantitative muscle testing;Diagnostic Test: Scale for Assessment and Rating of Ataxia;Diagnostic Test: Berg balance scale;Diagnostic Test: Quick motor function test;Diagnostic Test: GSGC;Diagnostic Test: 30 seconds sit to stand test;Diagnostic Test: Functional Index-2;Diagnostic Test: Six minute walking test;Diagnostic Test: Myotonia (from Individualised Neuromuscular Quality of Life Questionnaire) | Prof. Dr. Benedikt Schoser | NULL | Completed | 18 Years | 90 Years | All | 60 | Germany | |
| 2 | NCT02383511 (ClinicalTrials.gov) | February 2015 | 4/2/2015 | Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet | A Phase 1b Placebo-controlled, Multi-centre, Randomized, Double-blind Dose Escalation Study to Evaluate the Pharmacokinetics (PK) and Safety of SMT C1100 in Patients With Duchenne Muscular Dystrophy (DMD) Who Follow a Balanced Diet | Muscular Dystrophy, Duchenne | Drug: SMT C1100 | Summit Therapeutics | NULL | Completed | 5 Years | 13 Years | Male | 12 | Phase 1 | United Kingdom |
| 3 | EUCTR2014-003100-78-GB (EUCTR) | 01/12/2014 | 19/09/2014 | A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet. | SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet. | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: SMT C1100 INN or Proposed INN: Not available Other descriptive name: SMT C1100 | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 12 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom |