Clinical trials: Steinert's Disease
Myotonic Dystrophy 1, Dysphagia, Oropharyngeal
Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)
The goal of this observational study is to learn about swallowing difficulties (dysphagia) in patients living with myotonic dystrophy type 1 (DM1).
- Trial name
- SwallowDM1 (NCT05865483)
- Where
- United Kingdom
- Volunteers
- 177 people, healthy volunteers welcome
- Sponsor
- University College, London
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
Steinert's Disease
Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects
Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in subjects suffering from dystrophy would…
- Trial name
- NCT02930408
- Testing
- electrocardiogram, Echocardiography
- Volunteers
- 70 people
- Sponsor
- Poitiers University Hospital
Venous Thromboembolism, Pulmonary Embolism
Venous Thromboembolism in DM1
The risk for venous thromboembolism (VTE) in DM1 and in other inherited myopathies, which can lead to chronic immobilization, are unknown.
- Conditions
- Venous Thromboembolism, Pulmonary Embolism, Deep Vein Thrombosis, Myopathy
- Trial name
- DM1-VTE (NCT03141749)
- Volunteers
- 2,810 people
- Sponsor
- Institut de Myologie, France
Steinert Myotonic Dystrophy
Longitudinal Evaluation of Neuromuscular Involvement in Type 1 Myotonic Dystrophy
Primary objective: To determine the sensitivity to change of neuromuscular functional outcomes during the natural (non-interventional) progression of myotonic dystrophy type 1 (DM1), in order to identify the most relevant and…
- Trial name
- NatHis DM1 (NCT07362316)
- Testing
- Quantification of myotonia, MyoAnkle, Measurement of exercise-induced fatigue
- Where
- 2 sites in Canada, France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1)
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and…
- Conditions
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- NCT06809049
- Testing
- Music and movement education
- Where
- Canada
- Volunteers
- 13 people
- Sponsor
- Hanns Lochmuller
Quality of Life, Muscular Dystrophies
Quality of Life in Neuromuscular Disease
A Quality work was conducted to enable the future construction of a Quality of Life Questionnaire Related to Health (HRQoL) in patients with slowly progressive neuromuscular disease (NMD) as myopathies and muscular dystrophies.
- Trial name
- QoLNMD (NCT02895763)
- Testing
- Form administrations
- Where
- France
- Volunteers
- 159 people
- Sponsor
- CHU de Reims
Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)
Background: Myotonic dystrophy is a long-term genetic disorder that affects muscle function. Symptoms include gradually worsening muscle loss and weakness. Muscles often contract and cannot relax.
- Trial name
- NCT03867435
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Myotonic Dystrophy 1, Myotonic Dystrophy 2
Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study
The natural history of brain affection in myotonic dystrophy types 1 and 2 is still unknown. The investigators designed a 5-year longitudinal neuropsychological and neuroimaging follow-up study to address this issue.
- Trial name
- NCT02729597
- Testing
- medical history, neurological clinical examination, neuropsychological testing
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bonn
Myotonic Dystrophy 1, DM1
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and…
- Trial name
- NCT03981575
- Where
- 17 sites in United States, Canada and 5 more
- Volunteers
- 700 people
- Sponsor
- Virginia Commonwealth University
Myotonic Dystrophy 1
Home-based Training and Supplementation in DM1 Patients
Myotonic dystrophy type 1 (DM1) is a rare genetic disease that affects about 1 in 2100 people. Patients diagnosed with DM1 present with many symptoms, however, their muscles are mainly affected.
- Trial name
- DM1HBET (NCT05848830)
- Testing
- Mult-ingredient supplement, Concurrent exercise training
- Where
- Canada
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- McMaster University
Myotonic Dystrophy
Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients
Myotonic dystrophy Type 1 (MD1, Steinert's disease), an autosomal dominant multisystem disease, is of the most common muscular dystrophies in adults, with a European prevalence of 3-15/100 000.
- Trial name
- NCT01242007
- Testing
- Supine spirometry
- Where
- France
- Volunteers
- 58 people
- Sponsor
- University of Nancy
Myotonic Dystrophy Type 1 and Type 2
Study to Investigate the Efficacy and Safety of Mexiletine 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 Mexiletine During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 [The MIND Study]
- Trial name
- MIND (NCT04700046)
- Testing
- Mexiletine 167 mg
- Sponsor
- Lupin Ltd.
Myotonic Dystrophy
Extracellular RNA Biomarkers of Myotonic Dystrophy
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Trial name
- NCT05020002
- Where
- 3 sites in United States
- Volunteers
- 215 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
DM1, Muscular Dystrophies
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001…
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1
- Trial name
- MARINA-OLE (NCT05479981)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 37 people
- Sponsor
- Avidity Biosciences, Inc.
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
Cardiomyopathies, Amyloidosis Cardiac
Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
The study aims to identify new diagnostic and prognostic markers for CMP that can help predict disease progression. In particular, the study will focus on microRNAs (miRNAs) and spatial transcriptomics, which are emerging…
- Conditions
- Cardiomyopathies, Amyloidosis Cardiac, Fabry Disease, Arrhythmogenic Cardiomyopathy
- Trial name
- CMPMIRNA (NCT06813443)
- Where
- 3 sites in Italy
- Volunteers
- 700 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Myotonic Dystrophy Type 1
Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy
Study to focus on the defining and managing the neuropsychological abnormalities of myotonic dystrophy and to find out if the neuropsychological abnormalities have any correlation with changes seen on Magnetic Resonance Imaging.
- Trial name
- CHRI (NCT02269865)
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Stanford University
Myotonic Dystrophy 1
Effects of a 12-week Strength Training Program in Women With Myotonic Dystrophy Type 1
20 women with myotonic dystrophy type 1 (DM1) will complete a 12-week lower-limb strength training program. The training program consist of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg…
- Trial name
- NCT05400629
- Testing
- Training program
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- Université de Sherbrooke
Muscular Dystrophy, Arrhythmia
Arrhythmias in Myotonic Muscular Dystrophy
Adult myotonic muscular dystrophy (Steinert's disease) is the most common inherited neuromuscular disorder. Cardiac rhythm disturbances occur frequently in this disease state and may be responsible for up to one-third of deaths.
- Conditions
- Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
- Trial name
- DM1 (NCT00622453)
- Testing
- Screening
- Where
- United States
- Volunteers
- 448 people
- Sponsor
- Indiana University