Clinical trials: Congenital Myopathy
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
Psychoeducation, Parents
Psychoeducation Program for Parents of Children with Duchenne Muscular Dystrophy
Having and caring for a child with disabilities brings emotional, social and economic difficulties for many families. Families may experience many physiological and psychological problems due to the stress and anxiety they…
- Conditions
- Psychoeducation, Parents, Psychological Wellness, Duchenne Muscular Dystrophy
- Trial name
- NCT06412328
- Testing
- Psychoeducation
- Where
- Turkey (Türkiye)
- Volunteers
- 26 people
- Sponsor
- Lokman Hekim University
Cerebral Palsy, Down Syndrome
Investigation of Parents' Anxiety Level and Health Related Quality of Life in Different Types of Physical Disabilities
Disability brings many psychosocial problems in society. The effects of the health of a disabled child on the psychological health and quality of life of the family are inevitable.
- Conditions
- Cerebral Palsy, Down Syndrome, Spina Bifida, Muscular Dystrophy
- Trial name
- NCT04035967
- Testing
- Questionnaire
- Where
- Turkey (Türkiye)
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Sanko University
Muscular Dystrophies
Wearability, Saefty and Usability Assessment for the Upper Limb Exoskeleton BRIDGE/EMPATIA
The study is a feasibility study or pilot study, that is a clinical investigation to acquire the preliminary information on a motorized exoskeleton (BRIDGE / EMPATIA exoskeleton) for the movement of the upper limb in order to…
- Trial name
- NCT03951844
- Testing
- BRIDGE/EMPATIA exoskeleton
- Where
- 2 sites in Italy
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Alessandra Pedrocchi
Duchenne Muscular Dystrophy, Ambulation Difficulty
Postural and Anthropometric Properties of Foot and Ankle of Patients With DMD
Introduction: Progressive muscle weakness, joint contractures and body alignment disorders seen in patients with Duchenne Muscular Dystrophy (DMD) adversely affect the foot structure of the patients.
- Conditions
- Duchenne Muscular Dystrophy, Ambulation Difficulty, Posture Disorders in Children, Ambulation Disorder, Neurologic
- Trial name
- NCT05436210
- Testing
- foot posture index, north star ambulation assessment, timed performance tests, 6 minute walk test
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people
- Sponsor
- Hacettepe University
X-linked Myotubular Myopathy
A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger.
- Trial name
- INCEPTUS (NCT02704273)
- Where
- 8 sites in United States, Canada and 3 more
- Volunteers
- 34 people
- Sponsor
- Astellas Gene Therapies
Duchenne Muscular Dystrophy
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Primary Objective The primary objective of the study was to establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects.
- Trial name
- NCT02851797
- Testing
- givinostat
- Where
- 42 sites in United States, Belgium and 9 more
- Volunteers
- 179 people
- Sponsor
- Italfarmaco
Muscular Dystrophies, Limb-Girdle
A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants
This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
- Trial name
- COMPASS (NCT06952686)
- Testing
- SRP-9005, Corticosteroid
- Where
- United States
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy, Lower Urinary Tract Symptoms
Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy
The aim of this study is to examine the prevalence of lower urinary tract symptoms (LUTS) in children with Duchenne Muscular Dystrophy (DMD) and the relationship between functional level, posture, muscle strength, pelvic floor…
- Conditions
- Duchenne Muscular Dystrophy, Lower Urinary Tract Symptoms, Muscle Weakness, Posture Disorders in Children
- Trial name
- NCT05464446
- Testing
- Baseline Bubble Inclinometer, Baseline Digital İnclinometer and Hoggan microFET2 devices
- Where
- Turkey (Türkiye)
- Volunteers
- 45 people
- Sponsor
- Lokman Hekim University
Myotonic Dystrophy 1
Low-frequency Repetitive Nerve Stimulation in Myotonic Dystrophy Type 1
The study design is a prospective cohort study. It aims to evaluate the neuromuscular junction in dystrophic myotonia 1 (DM 1) using low-frequency repetitive nerve stimulation (RNS) on several nerve-muscle pairs of the one side…
- Trial name
- NCT05662150
- Testing
- Low frequency repetitive stimulation
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Vrije Universiteit Brussel
Duchenne or Becker Muscular Dystrophy
Effectiveness of Aquatic Therapy in Children With Duchenne and Becker Muscular Dystrophy
The purpose of this study is ; to evaluate the effects of aquatic therapy applied in addition to conventional physical therapy on balance, functionality and quality of life in children with Duchenne and Becker muscular dystrophy.
- Trial name
- NCT06186310
- Testing
- Aquatic Therapy Group, Conventional Physical Therapy Group
- Where
- Turkey (Türkiye)
- Volunteers
- 16 people
- Sponsor
- Gaziosmanpasa Research and Education Hospital
Myalgia, Hypercholesterolemia
Study of the Safety and Tolerability Associated With PPD10558 Versus Atorvastatin in Patients Previously Intolerant to Statins Due to Statin-associated Myalgia (SAM)
The purpose of this study is to assess the incidence of statin-associated myalgia (SAM) with treatment with PPD10558 versus atorvastatin in patients previously intolerant to statins.
- Conditions
- Myalgia, Hypercholesterolemia, Hyperlipidemia
- Trial name
- NCT01279590
- Testing
- PPD10558, Atorvastatin
- Where
- 67 sites in United States
- Volunteers
- 282 people
- Sponsor
- Furiex Pharmaceuticals, Inc
X-linked Myotubular Myopathy
A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07876804
- Testing
- ASP2957
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Muscular Dystrophy, Duchenne
A Study of Tadalafil for Duchenne Muscular Dystrophy
The main purpose of this study is to determine if tadalafil can slow the decline in walking ability of boys who have Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01865084
- Testing
- Tadalafil
- Where
- 62 sites in United States, Argentina and 14 more
- Volunteers
- 331 people
- Sponsor
- Eli Lilly and Company
Myotonic Dystrophy 1
Study of SRP-1003 in Participants 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 of single and multiple ascending doses of SRP-1003 compared to…
- Trial name
- NCT06138743
- Testing
- SRP-1003 IV Infusion, SRP-1003 SC Injection
- Where
- 35 sites in Australia, Belgium and 9 more
- Volunteers
- 78 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophies
Exercise Therapy for Patients with Muscle Dystrophies
Muscular Dystrophies (MD) are a heterogeneous group of diseases characterised by muscle wasting that lead to progressive loss of function.
- Trial name
- NCT05081791
- Testing
- Exercise Training
- Where
- Spain
- Volunteers
- 38 people
- Sponsor
- Universitat Pompeu Fabra
Duchenne Muscular Dystrophy (DMD)
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101.
- Trial name
- NCT06867107
- Testing
- SAT-3247
- Where
- 6 sites in United States, Australia
- Volunteers
- 30 people
- Sponsor
- Satellos Bioscience, Inc.
Muscular Dystrophies
A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- DMD114876 (NCT01462292)
- Testing
- GSK2402968 3mg/kg/week, GSK2402968 6 mg/kg/week
- Where
- 14 sites in United States
- Volunteers
- 51 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy
Physical Activity Level of Norwegian Boys with Duchenne Muscular Dystrophy
The aim of this population based study is to examine, quantify and describe physical activity level in Norwegian boys with DMD, and to compare the level of physical activity level between boys with DMD and age matched healthy…
- Trial name
- NCT03947112
- Testing
- Physical activity registration
- Where
- Norway
- Volunteers
- 28 people
- Sponsor
- Haukeland University Hospital
Telemedicine, Exercise
Feasibility of a Blended Therapy Approach
Exercise is an important part of therapy guidelines in the rehabilitation of rare diseases (RDs) as Haemophilia and Myositis. The aim of this study is not to evaluate a new therapy intervention, but to evaluate the delivery of…
- Conditions
- Telemedicine, Exercise, Feasibility, Myositis
- Trial name
- bletheRD (NCT03713151)
- Testing
- Dividat FIT
- Where
- Switzerland
- Volunteers
- 30 people
- Sponsor
- University of Zurich
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