Clinical trials: Dystrophia Myotonica 1
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
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 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 Type 1
Observational Prolonged Trial in Myotonic Dystrophy Type 1
Myotonic dystrophy type1 (DM1) is a rare, inherited, chronic progressive disease as well as an autosomal dominant multisystemic disorder.
- Trial name
- OPTIMISTIC (NCT02118779)
- Testing
- Behavioural change intervention
- Where
- 4 sites in France, Germany and 2 more
- Volunteers
- 255 people
- Sponsor
- Radboud University Medical Center
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
Myopathy
Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
Myotonic dystrophy type 1 is a myopathy with complex respiratory pattern and at risk to develop respiratory failure. Classical mode of ventilation are sometimes not tolerated or ineffective in this population.
- Trial name
- NCT01530841
- Testing
- Nocturnal ventilation
- Where
- France
- Volunteers
- 32 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Prostatic Neoplasms
MLN2704 in Subjects With Metastatic Androgen-Independent Prostate Cancer
The purpose of the study is to determine the highest dose of MLN2704 that can be given multiple times safely to patients with prostate cancer, and to identify any side effects associated with taking the drug.
- Trial name
- NCT00070837
- Testing
- MLN2704 (DM1 conjugated monoclonal antibody MLN591)
- Where
- 4 sites in United States
- Volunteers
- 46 people
- Sponsor
- Millennium Pharmaceuticals, Inc.
Dystrophia Myotonica Type 1, Myotonia Congenita
Lamotrigine as Treatment of Myotonia
Myotonia is a functional limiting symptom where the muscle stiffens on action leading to arrest of movement. Pharmacological treatment may make the difference between a physically restricted and a normal life.
- Conditions
- Dystrophia Myotonica Type 1, Myotonia Congenita, Paramyotonia Congenita, Hyperkalemic Periodic Paralysis
- Trial name
- NCT01939561
- Testing
- Lamotrigine
- Where
- Denmark
- Volunteers
- 27 people
- Sponsor
- Grete Andersen, MD
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
DM1, Myotonic Dystrophy
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Myotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy.
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1
- Trial name
- END-EXT (NCT07700225)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Virginia Commonwealth University
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
Myotonic Dystrophy 1
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy.
- Trial name
- NCT02858908
- Testing
- Tideglusib
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- AMO Pharma Limited
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
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
Type 1 Diabetes Mellitus
DM1, Effects of Carbo-Counting on Glycemic Control
Carbohydrate counting is the most effective meal-planning strategy in type 1 diabetes (T1DM) to optimize insulin therapy. However, it may lead to weight gain and unhealthy eating habits.
- Trial name
- NCT03554590
- Testing
- carbohydrate counting, insulin therapy according to standard care
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
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'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 Type 1
PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study)
PhenoDM1 will use patient reported outcomes to assess levels of pain, fatigue and quality of life in this cohort. Clinical and functional outcomes will look at muscle wasting and levels of myotonia.
- Trial name
- PhenoDM1 (NCT02831504)
- Where
- 2 sites in United Kingdom
- Volunteers
- 213 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
Myotonic Dystrophy 1, Sudden Cardiac Death
Sudden Cardiac Death Stratification in Myotonic Dystrophy Type 1 Patients
The aim of the study is to evaluate if the electrophysiological study (EPS) guided therapy, including the prophylactic implantation of implantable cardioverter defibrillator (ICD), in inducible patients, is able to improve…
- Trial name
- ACADEMY 1 (NCT03784586)
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- University of Campania Luigi Vanvitelli
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