113. 筋ジストロフィー
［臨床試験数：567，薬物数：442（DrugBank：93），標的遺伝子数：55，標的パスウェイ数：151］
Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT02858362 (ClinicalTrials.gov) | June 2016 | 27/7/2016 | Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD) | Phaseout DMD: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation With Ezutromid in Ambulatory Paediatric Male Subjects With Duchenne Muscular Dystrophy (SMT C11005) | Duchenne Muscular Dystrophy | Drug: Ezutromid | Summit Therapeutics | NULL | Terminated | 5 Years | N/A | Male | 43 | Phase 2 | United States;United Kingdom |
| 2 | EUCTR2015-004333-27-GB (EUCTR) | 19/01/2016 | 04/11/2015 | A clinical trial to test how the study medication (SMT C1100) works and how safe it is when given to children with Duchenne Muscular Dystrophy | A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005) - PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD | Duchenne Muscular Dystrophy MedDRA version: 20.0;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] | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 40 | Phase 2 | United States;United Kingdom | |||
| 3 | EUCTR2015-001967-38-GB (EUCTR) | 03/08/2015 | 20/07/2015 | A research project for boys with Duchenne muscular dystrophy aged 5-9 years. It aims to find out how safe and well tolerated different doses of the experimental medicine are, when given by mouth after breakfast and evening meal. | A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulation of SMT C1100 in Paediatric Subjects with Duchenne Muscular Dystrophy (DMD) - SMTC11004 - Phase 1 study in healthy volunteers and DMD patients | Duchenne muscular dystrophy (DMD) MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 1 | United Kingdom | |||
| 4 | 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 |
| 5 | 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] | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1 | United Kingdom | |||
| 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 |
| 6 | NCT02056808 (ClinicalTrials.gov) | November 2013 | 21/11/2013 | A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD) | SMT C1100 - A Phase 1b, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: SMT C1100 | Summit Corporation Plc. | NULL | Completed | 5 Years | 11 Years | Male | 12 | Phase 1 | United Kingdom |
| 7 | EUCTR2013-002115-99-GB (EUCTR) | 23/08/2013 | 31/07/2013 | A Clinical Trial to investigate if the drug SMT C1100 is safe and well tolerated in children with DMD when given orally one to three times a day. | SMT C1100 - A Phase 1, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.1;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 Name: SMT C1100 INN or Proposed INN: 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole | Summit Corporation plc | NULL | Not Recruiting | Female: no Male: yes | Phase 1 | United Kingdom |