114. 非ジストロフィー性ミオトニー症候群
［臨床試験数：10，薬物数：14（DrugBank：5），標的遺伝子数：18，標的パスウェイ数：10］
Searched query = "Non-dystrophic myotonia syndrome", "Non-dystrophic Myotonia", "Myotonia congenita", "Thomsen disease", "Becker disease", "Paramyotonia congenita", "Sodium channel myotonia"
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 | EUCTR2010-024026-38-NL (EUCTR) | 10/01/2012 | 09/02/2011 | Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia. - Mex vs. placebo in NDMs | Combining N-of-1 trials to estimate population clinical effectiveness of drugs using Bayesian hierarchical modeling. The case of Mexilitin for patients with Non-Dystrophic Myotonia. - Mex vs. placebo in NDMs | Patients with non-dystrophic myotonic syndromes (NDMs). NDMs are caused by mutations in the gene encoding for the skeletal muscle sodium (SCN4A) or chloride (CLCN1) channel. In this study patients with NDMs with a genetically confirmed mutation in the SCN4A-gene or the CLCN1-gene as registred in the Netherlands database of NDMs will be included. | Trade Name: Mexitil Product Name: mexitil INN or Proposed INN: MEXILETINE Other descriptive name: Mexitil | Radboud University Nijmegen Medical Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Netherlands | |||
| 2 | EUCTR2009-011184-36-IT (EUCTR) | 25/06/2010 | 05/09/2011 | Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - ND | Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - ND | Myotonic disorders are divided into dystrophic myotonias (DM1 and DM2) and non-dystrophic myotonias (NDM). The dystrophic myotonias are associated with significant progressive muscular weakness and other systemic organ involvement. On the other hand, NDM usually presents with muscle stiffness as the primary symptom, and severe weakness is not considered a major feature, especially in myotonia congenita. MedDRA version: 14.0;Level: LLT;Classification code 10032487;Term: Other specific muscle disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: MEXITIL*20CPS 200MG INN or Proposed INN: Mexiletine | Dept. of Neurology - Univ. of Kansas Medical Center | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United Kingdom;Italy |