114. Non-dystrophic myotonia syndrome
10 clinical trials,   14 drugs   (DrugBank: 5 drugs),   18 drug target genes,   10 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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agemin
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agemax
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PhaseCountries
1EUCTR2014-002627-10-IT
(EUCTR)
03/11/201505/11/2020Pivotal 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 BESTANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
58Phase 2Italy
2NCT02251457
(ClinicalTrials.gov)
August 201425/9/2014Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1Myotonia Congenita;Paramyotonia Congenita;Myotonic Dystrophy 1Drug: RanolazineOhio State UniversityGilead SciencesCompleted18 Years100 YearsAll35Phase 1United States
3NCT02045667
(ClinicalTrials.gov)
January 201418/11/2013Combined 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 MyotoniaDrug: Mexiletine;Drug: PlaceboRadboud UniversityZonMw: The Netherlands Organisation for Health Research and DevelopmentCompleted18 YearsN/ABoth30Phase 2Netherlands
4NCT01939561
(ClinicalTrials.gov)
November 201323/8/2013Lamotrigine as Treatment of MyotoniaLamotrigine as Treatment of Myotonia - a Phase 3 Randomized Controlled Trial StudyDystrophia Myotonica Type 1;Myotonia Congenita;Paramyotonia Congenita;Hyperkalemic Periodic Paralysis;Potassium-Aggravated MyotoniaDrug: Lamotrigine;Drug: PlaceboGrete Andersen, MDNULLCompleted18 YearsN/ABoth27Phase 3Denmark
5EUCTR2013-003309-24-DK
(EUCTR)
28/10/201321/10/2013Better treatment of muscle diseasesLamotrigine 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 AndersenNULLNot RecruitingFemale: yes
Male: yes
Denmark
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2010-024026-38-NL
(EUCTR)
10/01/201209/02/2011Combining 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 NDMsCombining 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 CenterNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Netherlands
7NCT02336477
(ClinicalTrials.gov)
June 20118/1/2015Mexiletine and Non Dystrophic MyotoniasEfficacy and Safety of Mexiletine in Non-dystrophic MyotoniasNon-dystrophic Myotonias;Paramyotonia Congenita;Myotonia CongenitaDrug: Mexiletine;Drug: placeboAssistance Publique - Hôpitaux de ParisNULLCompleted18 Years65 YearsBoth24Phase 3France
8EUCTR2009-011184-36-IT
(EUCTR)
25/06/201005/09/2011Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021) - NDPhase 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 CenterNULLNot RecruitingFemale: yes
Male: yes
60Phase 2United Kingdom;Italy
9NCT00832000
(ClinicalTrials.gov)
December 200827/1/2009Effectiveness of Mexiletine for Treating People With Non-Dystrophic MyotoniaPhase II Therapeutic Trial of Mexiletine in Non-Dystrophic MyotoniaMyotonia;Non-Dystrophic MyotoniaDrug: Mexiletine;Drug: PlaceboRichard Barohn, MDNULLCompleted16 YearsN/AAll59Phase 2United States;Canada;Italy;United Kingdom
10NCT00004802
(ClinicalTrials.gov)
June 199224/2/2000Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel DisordersParalysis, Hyperkalemic Periodic;Hypokalemic Periodic Paralysis;Paramyotonia CongenitaDrug: dichlorphenamideNational Center for Research Resources (NCRR)Ohio State UniversityCompleted10 Years75 YearsBoth64Phase 3NULL