Clinical trials: Steinert Disease
Myotonic Dystrophy 1, Muscular Dystrophies
Myotonic Dystrophy Type 1 Aerobic Exercise Study
Myotonic dystrophy type 1 (DM1) is a genetic disease that primarily targets skeletal muscle resulting in severe weakness and muscle loss.
- Trial name
- DM1ex (NCT04187482)
- Testing
- Exercise training
- Where
- Canada
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- McMaster University
Congenital Myotonic Dystrophy
Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy
Congenital Myotonic Dystrophy (CDM) is a multi-systemic, dominantly inherited disorder caused by a trinucleotide repeat expansion (CTGn) in the DMPK gene.
- Trial name
- TREAT-CDM (NCT03059264)
- Testing
- Natural history
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Virginia Commonwealth University
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
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 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
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Myotonic Dystrophy
Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)
The aim of this study is to investigate the safety and feasibility of daily subcutaneous injections of recombinant IGF1 complexed with IGF binding protein 3 (SomatoKine-INSMED) as a treatment for muscle wasting and weakness in…
- Trial name
- NCT00233519
- Testing
- SomatoKine/IPLEX
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University of Rochester
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
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
Myotonic Dystrophy 1, DM1
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents.
- Conditions
- Myotonic Dystrophy 1, DM1, Myotonic Dystrophy Type 1, Myotonic Dystrophy, Congenital
- Trial name
- DM1-Hub (NCT07385443)
- Testing
- Patient Registry
- Where
- 8 sites in Spain
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Fundació Institut Germans Trias i Pujol
Diabetes Mellitus, Type 1
Assess the Impact of Insulclock on Glycemic Variability and Treatment Compliance in Uncontrolled DM1 Patients
Insulclock® is a small electronic device developed to facilitate the optimal administration of insulin. This device works as an add-on module of commercially available insulin pens and monitors the date, time and dose of…
- Trial name
- Segoclock2 (NCT04847778)
- Testing
- Use of Insulclock system, both Insulclock device and Insulclock 360 app., Use of Insulclock system, both Insulclock device and Insulclock app on masked mode.
- Where
- 4 sites in Spain
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Hospital General de Segovia
Myotonic Dystrophy 1
Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy
The goal of this observational study is to assess movement in individuals with Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2) using digital biomarker tools.
- Trial name
- NCT06089018
- Where
- United States
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Stanford 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 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
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, Facioscapulohumeral Muscular Dystrophy
Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue.
- Conditions
- Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1
- Trial name
- NCT00082108
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1)
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders
The primary aim is to characterize the prevalence, severity and quality of musculoskeletal nociceptive pain in adult patients with neuromuscular disorders (NMD).
- Conditions
- Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- NCT04907162
- Testing
- Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory
- Where
- Germany
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- LMU Klinikum
Myotonic Dystrophy 1
Myotonic Dystrophy Type 1 and Resistance Exercise
The purpose of this study is to investigate the response after one bout of moderate-heavy resistance exercise in patients suffering from Myotonic Dystrophy type 1.
- Trial name
- STYRK DM1 (NCT05036447)
- Testing
- Moderate-Heavy Resistance Exercise
- Where
- Norway
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Norwegian School of Sport Sciences
DM1
Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients
The objective of the study is the modelisation of human cardiomyocyts from pluripotent stem cells in order to study the cardiac alterations induced by CTG expansions.
- Trial name
- NCT02084407
- Testing
- Clinical examination, skin biopsy, blood and urine sampling
- Where
- France
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
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