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Myotonic Dystrophy Type 1 clinical trials

View clinical trials related to Myotonic Dystrophy Type 1.

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NCT ID: NCT06316778 Enrolling by invitation - Clinical trials for Urinary Incontinence

Pelvic Floor Muscle Training for Women With Myotonic Dystrophy

Start date: March 11, 2024
Phase: N/A
Study type: Interventional

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). Urinary incontinence can occur in this situation and is often offset with compensatory measures without restoring the PFM function (e.g. sanitary pads). PFM training have already been shown to be effective in reducing or even eliminating urinary incontinence in the general population. However, no study has been the subject of this modality in people with DM1. Having recently shown that it is possible to gain strength with DM1, a strengthening protocol targeting PFM could prove effective in treating urinary incontinence. The objectives of this study are i) to assess the feasibility and acceptability of PFM training and ii) to investigate the effects of PFM training in women with DM1 with adult phenotype. A quasi-experimental study will be conducted with 12 women having a confirmed diagnosis of DM1 with urinary incontinence. Participants will follow a 12-week PFM training program, comprising weekly sessions with an experienced physiotherapist as well as a home exercise program. Outcomes measures will be assessed at baseline and at post-treatment and will include: feasibility and acceptability variables, frequency of urinary incontinence, urogynecological symptoms and their impact on quality of life, morphometry and function of PFM, and the perceived improvement following the treatments. This study has the potential to improve the management of urinary incontinence and support the implementation of pelvic floor rehabilitation services in this population.

NCT ID: NCT06075693 Recruiting - Clinical trials for Myotonic Dystrophy Type 1

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

Start date: August 1, 2022
Phase:
Study type: Observational

Myotonic dystrophy is associated with central sleep apnea, excessive daytime sleepiness, diminished working memory, impaired visuospatial skills, and deficits in problem-solving skills. Cerebrospinal fluid (CSF) is a clear, colorless fluid that surrounds and protects the brain. Changes in the composition of CSF can serve as early indicators of changes in brain activity and function. The purpose of this research is to learn about myotonic dystrophy by examining cerebrospinal fluid and brain activity in participants. The tests will be low risk and are well tolerated. The information that we gather from this study may help us evaluate, prevent, diagnose, treat, and improve our understanding of myotonic dystrophy

NCT ID: NCT05916677 Enrolling by invitation - Clinical trials for Myotonic Dystrophy Type 1

Virtual Reality-training in Theory of Mind in the Childhood Form of Myotonic Dystrophy Type 1

TOM-VR-DM1
Start date: October 30, 2023
Phase: N/A
Study type: Interventional

The ability of an individual to conceive some alternative representations and to behave in a flexible manner would emerge from preschool age and drastically improve between the ages of 3 and 5 (Doebel and Zelazo, 2013). They constitute, according to Diamond (2013), a prerequisite for the development of the Theory of Mind (ToM). Deficits in Executive Functions (EF) may therefore interfere with the child's ability to understand and adapt to social situations. Treatment failures are often observed when traditional cognitive tools are used. This would be linked to the divergence between non-immersive tests and situations encountered in everyday life (Damasio, 1994; Priore Castelnuovo and Liccione, 2002). For this reason, an increasing number of researchers are using virtual reality for the rehabilitation of executive functions and Theory of Mind in patients with neurodevelopmental pathology (Millen, Edlin-White and Cobb, 2010) or brain damage (Le Gall, Besnard, Louisy, Richard and Allain 2008). There is currently no systematic evaluation of ToM in children with the infantile form of DM1 even though these abilities are considered particularly vulnerable and have a decisive impact on the subsequent development of interpersonal relationships. This research will focus on studying the socio-emotional disorders associated with the infantile form of Myotonic Dystrophy type 1 (DM1). The axis that we propose to develop more specifically will be an interventional study with the aim of remediation with children from 5 to 16 years old suffering from the infantile form of DM1 via a training protocol in low-immersion Virtual Reality (VR) centered on emotional processing and theory of mind.

NCT ID: NCT03867435 Terminated - Clinical trials for Myotonic Dystrophy Type-2

Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

Start date: February 24, 2020
Phase:
Study type: Observational

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. Researchers want to find out how various tests for DM1 or DM2 change over 2 years, to help them develop better tests for people with these diseases. Data and samples from this study will be shared with the Myotonic Dystrophy Clinical Research Network (DMCRN) investigators participating in the ongoing Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (ENDDM1) study Objective: To find better ways to assess how myotonic dystrophy type 1 or type 2 affects people. Eligibility: People ages 11 70 with DM1 or DM2 Design: Participants will have 3 study visits over 2 years. Participants may be admitted to the clinic. Each visit may last up to a week and will include: Medical history and physical exam Blood, heart, and pregnancy tests Questions about their disease Breathing and muscle tests, including tests of movement, grip, and hand opening Speech and swallowing exam Magnetic resonance imaging (MRI). Participants will lie on a table that slides into a cylinder. A magnetic field and radio waves will take pictures of the body. They may do a task during the scan. They may have a dye injected. Pictures of chemicals in the brain or muscle taken in an MRI scanner Thinking and memory tests Sleep studies. Electrodes placed on the scalp will record the electrical activity of the brain. Other devices on the body will measure heartbeat, breathing, movement, and oxygen. Tests of electrical activity of muscles. Participants move their arms and legs with disks stuck on their skin. Visits may also include: Exam by a physician expert in stomach and bowel disorders A piece of muscle and/or spinal fluid removed by needle Sponsoring Institute: National Institute of Neurological Disorders and Stroke ...

NCT ID: NCT02831504 Completed - Clinical trials for Myotonic Dystrophy Type 1

PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study)

PhenoDM1
Start date: August 2015
Phase:
Study type: Observational

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. DNA, RNA, serum and CSF samples will be taken from all patients so that additional genetic and molecular biomarker analysis can be carried out. A subset of patients will undergo detailed sleep studies along with skeletal muscle MRI of the lower limbs. This study will complement the work of other groups currently looking at myotonic dystrophy type 1 using the same outcomes and measures where possible.

NCT ID: NCT02312011 Completed - Clinical trials for Myotonic Dystrophy Type 1

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

Start date: December 2014
Phase: Phase 1/Phase 2
Study type: Interventional

This study will test the safety, tolerability, and pharmacokinetics of multiple escalating doses of ISIS-DMPKRx administered subcutaneously to adult patients with DM1.

NCT ID: NCT02308657 Completed - Clinical trials for Myotonic Dystrophy Type 1

Multicenter Observational Study of Myotonic Dystrophy Type 1

MOS-DM1/POP
Start date: November 2013
Phase: N/A
Study type: Observational

The purpose of the study is to determine the best ways to assess how people are affected by myotonic dystrophy type 1 (DM1). The study will assess walking speed, muscle strength, muscle size, myotonia, heart rhythm, mental efficiency, and overall health. Participants will complete questionnaires to record their ideas about how they are affected by DM1. The study will evaluate people with DM1 over 1 year to determine how the condition changes over time. The study will identify biomarkers of DM1. Biomarkers are laboratory measurements that show the effects of DM1 on a person's muscle tissue or blood. Biomarkers are needed in future studies to determine how DM1 may respond to treatments.

NCT ID: NCT02269865 Recruiting - Clinical trials for Myotonic Dystrophy Type 1

Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy

CHRI
Start date: December 2013
Phase: N/A
Study type: Observational

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.

NCT ID: NCT02118779 Completed - Clinical trials for Myotonic Dystrophy Type 1

Observational Prolonged Trial in Myotonic Dystrophy Type 1

OPTIMISTIC
Start date: April 2, 2014
Phase: N/A
Study type: Interventional

Myotonic dystrophy type1 (DM1) is a rare, inherited, chronic progressive disease as well as an autosomal dominant multisystemic disorder. It is the most common adult form of muscular dystrophy, with a prevalence of approximately 10 per 100,000 people affected. With 733 million people in Europe, we estimate that 75,000 people are DM1 patients in Europe. The aim of OPTIMISTIC is to improve clinical practice in the management of patients with this rare disease for which no dedicated treatment is currently available. OPTIMISTIC is a multi-centre, randomised controlled trial designed to compare a two component tailored behavioural change intervention to increase physical activity against standard patient management regimes, with particular attention given to the definition of appropriate outcome measures and new clinical guidelines for DM1 management. The two components of the intervention are 1) cognitive behavioural therapy (CBT) and 2) graded physical activity and we will evaluate the intervention's effectiveness and safety against standard patient management. Participants will be recruited from myotonic dystrophy clinics and neuromuscular centres in France, Germany, the Netherlands and the UK. A total of 286 male and female patients aged 18 years and older with genetically proven classical or adult DM1 suffering from severe fatigue (only DM1 patients with a CIS subscale fatigue score > 35 are likely to benefit from the intervention), able to walk independently and able to complete the trial interventions will be included. A key objective of OPTIMISTIC is to provide outcome measures that are relevant for the patients and have a rate of change that is appropriate for a clinical trial timeframe. In addition, OPTIMISTIC will identify genetic factors that predict outcome and potential biomarkers as surrogate outcome measures that best explain the observed clinical variation.

NCT ID: NCT00577577 Completed - Clinical trials for Myotonic Dystrophy Type 1

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Start date: December 2007
Phase: Phase 2
Study type: Interventional

To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients