Clinical trials: Myotonic Dystrophy 2
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
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
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 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
Myotonic Dystrophy 1
Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
Myotonic Dystrophy type 1 (DM1) is a multisystem disease that causes muscle weakness and myotonia. As a result upper limb function might become impaired.
- Trial name
- SOUL-DM1 (NCT05006924)
- Where
- 3 sites in Norway
- Volunteers
- 89 people
- Sponsor
- Oslo University Hospital
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
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 Type 1, Urinary Incontinence
Pelvic Floor Muscle Training for Women with Myotonic Dystrophy
Myotonic dystrophy type 1 (DM1) is a neuromuscular disease characterized by multisystem manifestations. DM1 can affect the urinary system through the impact of the pelvic floor muscles (PFM).
- Trial name
- NCT06316778
- Testing
- Pelvic floor muscle training
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- Université de Sherbrooke
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
Open Label Study in Adolescents and Children With Myotonic Disorders
This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to <18 years of age) with myotonic disorders.
- Trial name
- NCT04624750
- Testing
- Mexiletine
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Lupin Ltd.
Myotonic Dystrophy, Congenital Myotonic Dystrophy
Myotonic Dystrophy Family Registry
The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in developing new, effective treatments and help identify participants…
- Conditions
- Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy 2
- Trial name
- MDFR (NCT02398786)
- Testing
- Patient-entered data
- Where
- United States
- Volunteers
- 3,500 people
- Sponsor
- Myotonic Dystrophy Foundation
Inclusion Body Myositis, Myotonic Dystrophy
The Risk of Falls Index for Patients With Neuromuscular Disorders
The combination of short quantitatively assessing muscular function and balance in combination with short clinical scores, can be a new valid approach to evaluate the patient risk of fall and help to create a quick checkup test…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophies, Myasthenia Gravis
- Trial name
- NCT05890833
- Testing
- The risk of falls
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Wheelchair Mobility, Manual Wheelchair Skills Training
Wheelchair Skills Training for People with ARSACS and DM1
Wheelchairs (WC) are often provided to people with ARSACS and MD1 when they are not able to walk anymore. However, giving someone a MWC alone does not guarantee they will use it safely or properly.
- Conditions
- Wheelchair Mobility, Manual Wheelchair Skills Training, ARSACS, Myotonic Dystrophy Type 1
- Trial name
- NCT06596850
- Testing
- Wheelchair Skills Training Program
- Where
- Canada
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Laval University
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
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
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 1
Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]
The rationale of the study is to collect structured data in the neuropsychological, clinical neuroradiologic and neurorehabilitation fields in children/young people affected by congenital and juvenile myotonic dystrophy.
- Trial name
- MDCJ-NeuBeRe (NCT06378216)
- Testing
- neurocognitive evaluations
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Myotonic Dystrophy 1
Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach
Type 1 myotonic dystrophy (MD1) is a genetic and hereditary disease that primarily affects muscle tissue, resulting in myotonia (difficulty relaxing after contraction) and atrophy (progressive muscle weakening with decreased…
- Trial name
- GoodDiagNMD (NCT06270186)
- Testing
- Type 1 myotonic dystrophy
- Sponsor
- Centre Hospitalier Universitaire de Nice
Myotonic Dystrophy 1
Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
Impairment of balance and gait are frequent complaints in patients with myotonic dystrophy type 1 (DM1). In these persons, there is an increased risk for stumbles and falls when compared to normal subjects.
- Trial name
- CABLAMYD (NCT04712422)
- Testing
- Healthy subjects, Pathologic group
- Where
- 2 sites in Italy
- Volunteers
- 42 people
- Sponsor
- Istituto Auxologico Italiano