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Muscular Dystrophy Clinical Trials

A listing of Muscular Dystrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 135 clinical trials

Clinical Evaluation of the Medical Device (MD) (Caloprothese Connected Kit, CCK)

Neuropathic pain is common in limb amputees and causes reductions in activity and participation as well as impaired quality of life. Some of these pains lead to the diagnosis of a responsible lesion and to precise and effective treatments (amputation neuroma pains, for example), whether they are etiological or symptomatic. …

18 years of age All Phase N/A
Z Zoe Sheitman, DPT

Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

An exercise regimen (PRIME: Proximal Resistance In-House Movement Exercise) has been designed for patients with myotonic dystrophy type 2 (DM2). The hypothesis is that this patient-friendly physical therapist (PT)-guided exercise program associates with improved functional capacity and muscle composition in DM2 in this two-period two-sequence cross-over study. Thus, participant will …

18 - 70 years of age All Phase N/A
W Woo Hyung Lee, MD, Ph.D

The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder

The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder. The main questions it aims to answer are: What is the impact of the muscle-mimicking, fabric-type shoulder orthosis on upper limb …

10 years of age All Phase N/A
R Romain FEIGEAN, PhD

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. The proliferation of clinical trials has created a need to identify biomarkers that are both sensitive to changes and specific to the disease. Current gait tests only consider the time factor and …

18 - 65 years of age All Phase N/A
H Hanne L Fossmo, MSC

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: Charcot-Marie-Tooth (CMT), Facioscapulohumeral Muscular Dystrophy (FSHD), and Myotonic Dystrophy Type 1 (DM1). The key objectives are: To investigate if the …

18 - 70 years of age All Phase N/A
F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders

Background People with cerebral palsy, spina bifida, muscular dystrophy, or spinal cord injury often have muscle weakness and problems controlling how their legs move. This can affect how they walk. The NIH has designed a robotic device (exoskeleton) that can be worn on the legs while walking. The wearable robot …

3 - 17 years of age All Phase N/A

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1

This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 50 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in …

10 - 50 years of age All Phase 1/2
P Patient Advocacy

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 to placebo.

16 - 60 years of age All Phase 2

Study of ATX-01 in Participants With DM1

The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo. There will be …

18 - 64 years of age All Phase 1/2

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

This is a Phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects with Type 1 Myotonic Dystrophy (DM1). Participants who have provided written informed consent and met …

18 - 65 years of age All Phase 1

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