Clinical trials: Congenital Myotonic Dystrophy, active
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
Myopathy, Muscular Dystrophies
Muscle Health Measurements Using Electrical Impedance Myography
This study is being done to further develop a device, the mScan, to measure muscle health as compared to measurements of muscle health using MRI (magnetic resonance imaging).
- Conditions
- Myopathy, Muscular Dystrophies, Myositis, Myofibrillar Myopathy
- Trial name
- NCT07502989
- Testing
- Electrical Impedance Myography
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
Steinert's Disease, Myotonic Dystrophy 1
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
The study team hypothesize that non-diabetic patients with Myotonic dystrophy type I (DM1) will improve their symptoms, especially their motor deficit which is the main feature of the disease, because of the splicing defect…
- Conditions
- Steinert's Disease, Myotonic Dystrophy 1, Metformin
- Trial name
- METFORMYO (NCT05532813)
- Testing
- Treatment taken
- Where
- France
- Volunteers
- 142 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
Nearly two-third of patients with myotonic dystrophy type 2 (DM2) report that impaired cognition is among the most disabling symptoms and deeply affects their quality of life.
- Trial name
- BraCE-DM2 (NCT05854433)
- Testing
- Non-interventional study
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases.
- Conditions
- Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Congenital Myopathy
- Trial name
- ARNseq-Musc (NCT06833489)
- Testing
- ARN extraction from muscle biopsies
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
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 1
Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)
Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystem disorder caused by an expanded CTG trinucleotide repeat in the 3' untranslated region of the DMPK gene.
- Trial name
- C-DMCOS-DM1 (NCT06101940)
- Testing
- MRI scan, Electrocardiography, Pulmonary function test
- Where
- 22 sites in China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Huashan Hospital
Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases.
- Conditions
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies, Healthy Participants
- Trial name
- Dystro-miR1 (NCT07415837)
- Testing
- dosage of blood biomarker miR1
- Where
- France
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
Myotonic Dystrophy 1, Myotonic Dystrophy 2
An Ambispective Natural History Study in Myotonic Dystrophy Patients Linking Retrospective Data Captured From the DM-Scope Registry With a Prospective 24-month Follow-up Period
This natural history observational study is being conducted to follow patients with DM1 or DM2 over a 2 year period to study the presence of myotonia, how it's perceived and its impact on patients quality of life.
- Trial name
- Track-DM (NCT07732439)
- Where
- 6 sites in France
- Volunteers
- 100 people
- Sponsor
- Lupin Ltd.
Myotonic Dystrophy Type 1, Congenital DM1
Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy
Myotonic dystrophy type 1 (DM1) can affect people in many different ways, even in the same family. The symptoms that children experience can be different and more severe than adults.
- Conditions
- Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
- Trial name
- REACH-DM KIDS (NCT07630389)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Rochester
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.
- Conditions
- Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
- Trial name
- TREAT-EXT (NCT06747884)
- Where
- 4 sites in United States, Brazil
- Volunteers
- 200 people
- Sponsor
- Virginia Commonwealth University
Myotonic Dystrophy Type 1 (DM1)
A Remote Physical Activity Program in the Population Suffering from Type 1 Myotonic Dystrophy
The COVID-19 pandemic exacerbates health problems by reducing access to adapted and advanced physical rehabilitation for several people who need rehabilitation services, including the population with myotonic dystrophy type 1…
- Trial name
- NCT05072288
- Testing
- Remote activity program
- Where
- 2 sites in Canada
- Volunteers
- 40 people
- Sponsor
- Université du Québec à Chicoutimi
Central Core Disease, Centronuclear Myopathy
Molecular and Genetic Studies of Congenital Myopathies
In the Congenital Myopathy Research Program at Boston Children's Hospital and Harvard Medical School, the researchers are studying the congenital myopathies (neuromuscular diseases present from birth), including central core…
- Conditions
- Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease
- Trial name
- NCT00272883
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Boston Children's Hospital
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)
A Registered Observational Cohort Study of Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy.There is little phenotype and genetic data for Chinese DM1 patients.
- Trial name
- NCT06979024
- Testing
- Triplet-primed PCR or Long-read sequencing
- Where
- China
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device.
- Conditions
- Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth
- Trial name
- NCT05982119
- Testing
- ActiMyo/Syde
- Where
- 8 sites in Belgium, Czechia and 5 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Myotonic Dystrophy
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES…
- Trial name
- NCT06523400
- Testing
- Mexiletine granules for prolonged-release oral suspension
- Where
- 7 sites in Belgium, Denmark and 4 more
- Volunteers
- 176 people
- Sponsor
- Lupin Ltd.