114. 非ジストロフィー性ミオトニー症候群 Non-dystrophic myotonia syndrome Clinical trials / Disease details
臨床試験数 : 12 / 薬物数 : 19 - (DrugBank : 5) / 標的遺伝子数 : 18 - 標的パスウェイ数 : 10
| 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 | EUCTR2021-003784-94-DK (EUCTR) | 20/01/2022 | 30/11/2021 | Which treatment are the best to reduce myotonia? | Treatment of Myotonia - Lamotrigine versus Namuscla | Myotonia Congenita (Becker and Thomsen type), Paramyotonia Congenita, and Hyperkalemic periodic paralysis MedDRA version: 20.1;Level: LLT;Classification code 10049841;Term: Myotonia congenital;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10076440;Term: Hyperkalemic periodic paralysis;System Organ Class: 100000004850;Therapeutic area: Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutic techniques [E02] | Trade Name: Namuscla INN or Proposed INN: Mexiletine Trade Name: Lamotrigin ”Nordic Prime 25 mg INN or Proposed INN: LAMOTRIGINE Trade Name: Lamotrigin ”Nordic Prime 50 mg INN or Proposed INN: LAMOTRIGINE Trade Name: Lamotrigin ”Nordic Prime 100 mg INN or Proposed INN: LAMOTRIGINE | Rigshospitalet, department of Neurology | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 3 | Denmark | ||
| 2 | NCT05017155 (ClinicalTrials.gov) | August 12, 2021 | 11/8/2021 | MExiletine Versus Lamotrigine in Non-Dystrophic Myotonias | A Phase III, Randomised, Double Blinded, Head-to-head, Single-site, Cross-over Trial of Lamotrigine Versus Mexiletine for Non-dystrophic Myotonias | Non-Dystrophic Myotonia | Drug: Mexiletine;Drug: Lamotrigine 25Mg Oral Tablet, Extended Release | University College, London | NULL | Recruiting | 18 Years | 90 Years | All | 60 | Phase 3 | United Kingdom |
| 3 | EUCTR2014-002627-10-IT (EUCTR) | 03/11/2015 | 05/11/2020 | Pivotal study to evaluate the efficacy and safety of riluzole versus mexiletine in patients with non dystrophic myotonia mutated in SCN4A orCLCN1 genes. | A randomized, double-blind, controlled, monocenter, pivotal phase IIb study to evaluate the efficacy and safety of riluzole versus mexiletine in patients with non dystrophic myotonia mutated in SCN4A or CLCN1 genes. - RILUMEX | NDM patients with mutations in SCN4A or CLCN1 gene MedDRA version: 20.0;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: RILUZOLO TEVA - 50 MG COMPRESSE RIVESTITE CON FILM 90 COMPRESSE IN BLISTER AL/PVC Product Name: RILUZOLO INN or Proposed INN: RILUZOLO INN or Proposed INN: MEXILETINA CLORIDRATO | FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 58 | Phase 2 | Italy | ||
| 4 | NCT02251457 (ClinicalTrials.gov) | August 2014 | 25/9/2014 | Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1 | Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1 | Myotonia Congenita;Paramyotonia Congenita;Myotonic Dystrophy 1 | Drug: Ranolazine | Ohio State University | Gilead Sciences | Completed | 18 Years | 100 Years | All | 35 | Phase 1 | United States |
| 5 | NCT02045667 (ClinicalTrials.gov) | January 2014 | 18/11/2013 | Combined N-of-1 Trials Mexiletine vs Placebo in Patients With Non-Dystrophic Myotonia (NDM) | Combining N-of-1 Trials to Estimate Population Clinical and Cost-effectiveness of Drugs Using Bayesian Hierarchical Modeling. The Case of Mexiletine for Patients With Non- Dystrophic Myotonia. | Non Dystrophic Myotonia | Drug: Mexiletine;Drug: Placebo | Radboud University | ZonMw: The Netherlands Organisation for Health Research and Development | Completed | 18 Years | N/A | Both | 30 | Phase 2 | Netherlands |
| 6 | NCT01939561 (ClinicalTrials.gov) | November 2013 | 23/8/2013 | Lamotrigine as Treatment of Myotonia | Lamotrigine as Treatment of Myotonia - a Phase 3 Randomized Controlled Trial Study | Dystrophia Myotonica Type 1;Myotonia Congenita;Paramyotonia Congenita;Hyperkalemic Periodic Paralysis;Potassium-Aggravated Myotonia | Drug: Lamotrigine;Drug: Placebo | Grete Andersen, MD | NULL | Completed | 18 Years | N/A | Both | 27 | Phase 3 | Denmark |
| 7 | EUCTR2013-003309-24-DK (EUCTR) | 28/10/2013 | 21/10/2013 | Better treatment of muscle diseases | Lamotrigine as treatment of Myotonia | Myotonia is a symptom seen certain inherited muscle dystrophies and pure channelopathies (Dystrophia myotonica type 1 and 2, Myotonia Congenita, Paramyotonia Congenita, Hyperkalæmisk periodic paralysis and Potassium-Aggravated myotonia). Genetically, mutations in chloride, sodium or calcium ion channels in the muscle is present. Myotonia manifests during physical activity where the muscle can locks in the middle of a movement. Patients are limited in their physical as well as social activities. MedDRA version: 14.1;Level: PT;Classification code 10061533;Term: Myotonia;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Lamotrigin actavis INN or Proposed INN: Lamotrigine Other descriptive name: LAMOTRIGINE | Grete Andersen | NULL | Not Recruiting | Female: yes Male: yes | Denmark | ||||
| 8 | 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 | |||
| 9 | NCT02336477 (ClinicalTrials.gov) | June 2011 | 8/1/2015 | Mexiletine and Non Dystrophic Myotonias | Efficacy and Safety of Mexiletine in Non-dystrophic Myotonias | Non-dystrophic Myotonias;Paramyotonia Congenita;Myotonia Congenita | Drug: Mexiletine;Drug: placebo | Assistance Publique - Hôpitaux de Paris | NULL | Completed | 18 Years | 65 Years | Both | 24 | Phase 3 | France |
| 10 | 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 | ||
| 11 | NCT00832000 (ClinicalTrials.gov) | December 2008 | 27/1/2009 | Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia | Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia | Myotonia;Non-Dystrophic Myotonia | Drug: Mexiletine;Drug: Placebo | Richard Barohn, MD | NULL | Completed | 16 Years | N/A | All | 59 | Phase 2 | United States;Canada;Italy;United Kingdom |
| 12 | NCT00004802 (ClinicalTrials.gov) | June 1992 | 24/2/2000 | Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders | Paralysis, Hyperkalemic Periodic;Hypokalemic Periodic Paralysis;Paramyotonia Congenita | Drug: dichlorphenamide | National Center for Research Resources (NCRR) | Ohio State University | Completed | 10 Years | 75 Years | Both | 64 | Phase 3 | NULL |