Clinical trials: Myotonic Dystrophy
Glycogen Storage Disease Type 2, Dystrophia Myotonica
An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-
The aim of the project is to develop new Magnetic Resonance (MR) imaging techniques for better diagnosis and monitoring of patients with muscular disorders.
- Trial name
- NCT02708784
- Where
- 2 sites in Denmark
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
Myotonic Dystrophy Type 1 (DM1)
Technology Assisted Rehabilitation for Upper Limb Function in Myotonic Dystrophy Type 1
Myotonic Dystrophy type 1 (DM1) is a genetic multisystem disease causing muscle weakness and myotonia. As a result, upper limb function might become impaired.
- Trial name
- Technorehab (NCT05560438)
- Testing
- Tyromotion Amadeo and Armeo Senso
- Where
- Norway
- Volunteers
- 6 people
- Sponsor
- University of Oslo
Muscular Dystrophies, Congenital Myopathy
Assessment of Safety and Acute Effects of a Knee-hip Powered Soft Exoskeleton in Patients With Neuromuscular Disorders
The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a powered knee-hip dermoskeleton (MyoSuit, MyoSwiss, Zurich, Switzerland) in patients with neuromuscular disorders, ii)…
- Conditions
- Muscular Dystrophies, Congenital Myopathy, Idiopathic Inflammatory Myopathies, Mitochondrial Myopathies
- Trial name
- Exo-NMD1 (NCT05200702)
- Testing
- Knee-hip Powered Soft Exoskeleton (MyoSuit, MyoSwiss, Zurich, Switzerland)
- Where
- France
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Neuromuscular Diseases
Muscle Oxygenation in Effort in Neuromuscular Diseases
Previous studies showed modifications of muscle oxygenation parameters in muscular dystrophies du to an impairment or an absence of dystrophin.
- Trial name
- OXYNEMU (NCT02789059)
- Testing
- muscle oxygenation
- Where
- 3 sites in France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University Hospital, Lille
Myotonic Dystrophy, Sudden Cardiac Death
RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy
This is a prospective multicentric Italian study to evaluate the arrhythmic risk in myotonic dystrophy type 1.
- Trial name
- NCT00127582
- Testing
- Electrophysiological study, pacemaker , implantable cardiac defibrillator or loop-recorder implant
- Where
- Italy
- Volunteers
- 537 people
- Sponsor
- Catholic University of the Sacred Heart
Myotonic Muscular Dystrophy
Study of Muscle Wasting and Altered Metabolism in Patients With Myotonic Dystrophy
OBJECTIVES: I. Examine the interrelationships between muscle wasting (phenotype), the degree of myotonic dystrophy (DM) gene expression (genotype) in patients with DM. II. Characterize the insulin resistance in these patients.
- Trial name
- NCT00004769
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Myotonic Dystrophy Type 1 (DM1)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- ACHIEVE (NCT05481879)
- Testing
- DYNE-101
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 127 people
- Sponsor
- Dyne Therapeutics
Myotonic Dystrophy
An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM.
- Trial name
- NCT04616807
- Testing
- Mexiletine
- Where
- 6 sites in France, Germany and 1 more
- Volunteers
- 53 people
- Sponsor
- Lupin Ltd.
Myotonic Dystrophy Type 1, Falls
Fall Frequency and Factors Affecting Dynamic Balance in Patients With Myotonic Dystrophy Type 1
This cross-sectional observational study aims to determine the frequency of falls in patients with myotonic dystrophy type 1 and to identify factors affecting dynamic balance.
- Trial name
- NCT07493525
- Where
- Turkey (Türkiye)
- Volunteers
- 41 people
- Sponsor
- Antalya Training and Research Hospital
Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion. The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants…
- Trial name
- NCT03959189
- Testing
- ERX-963
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Expansion Therapeutics, Inc.
Nemaline Myopathy Type 6, Myotonic Dystrophy Type 2
Muscle Relaxation in Myopathies With Positive Muscle Phenomena
The aim of this study is to quantify muscle relaxation properties of the finger flexor muscles in patients with different myopathies.
- Conditions
- Nemaline Myopathy Type 6, Myotonic Dystrophy Type 2, McArdle Disease
- Trial name
- NCT03211923
- Testing
- Transcranial magnetic stimulation (TMS)
- Volunteers
- 30 people
- Sponsor
- Radboud University Medical Center
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Congenital Myotonic Dystrophy
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
- Trial name
- NCT03692312
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 56 people
- Sponsor
- AMO Pharma Limited
Myotonic Dystrophy 1
Calcium Channel Blocker in Myotonic Dystrophy Type 1
This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of amlodipine, a calcium channel blocker, in adults with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- CAP DM1 (NCT07075965)
- Testing
- Amlodipine
- Volunteers
- 20 people
- Sponsor
- University of Rochester
Congenital Myotonic Dystrophy, CDM
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.
- Trial name
- ASPIRE-DM1 (NCT05224778)
- Where
- 5 sites in United States, Italy
- Volunteers
- 50 people
- Sponsor
- Virginia Commonwealth University
Myotonia Congenita, Paramyotonia Congenita
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1.
- Conditions
- Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
- Trial name
- NCT02251457
- Testing
- Ranolazine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Ohio State University
Myotonic Dystrophy 1
Low-frequency Repetitive Nerve Stimulation in Myotonic Dystrophy Type 1
The study design is a prospective cohort study. It aims to evaluate the neuromuscular junction in dystrophic myotonia 1 (DM 1) using low-frequency repetitive nerve stimulation (RNS) on several nerve-muscle pairs of the one side…
- Trial name
- NCT05662150
- Testing
- Low frequency repetitive stimulation
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Vrije Universiteit Brussel
CDM, ChDM
Trial Readiness and Endpoint Assessment in Congenital and Childhood Myotonic Dystrophy
Children with congenital myotonic dystrophy (CDM) present at birth with respiratory insufficiency, talipes equinovarus, feeding difficulties and hypotonia. There is a 30% mortality rate in the first year of life.
- Trial name
- GUP19002 (NCT06276244)
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- Fondazione Serena Onlus - Centro Clinico NeMO Milano