Clinical trials: Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
Background and Rationale Duchenne muscular dystrophy (DMD) is a genetic disorder caused by mutations in the dystrophin gene, resulting in progressive muscle weakness and multiple complications, including respiratory and cardiac…
- Trial name
- GUP23005 (NCT07890883)
- Where
- 15 sites in Italy
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Istituto Giannina Gaslini
Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA)
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy
The goal of this clinical trial is to learn if providing a multicomponent intervention improves the confidence of caregivers of children with DMD or SMA.
- Trial name
- SEE-NMD (NCT07356063)
- Testing
- Multicomponent Intervention
- Where
- Pakistan
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Aga Khan University
Muscular Dystrophy, Peripheral Neuropathy
Investigational Use of Neuromuscular Ultrasound
Background: Current techniques used to measure the health and function of a person s nerves and muscles are generally effective, but they do have limits.
- Conditions
- Muscular Dystrophy, Peripheral Neuropathy, Motor Neuron Disorder, Normal Physiology
- Trial name
- NCT05237973
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Duchenne Muscular Dystrophy (DMD), Lower Urinary Tract Symptoms (LUTS)
Urotherapy Education for Families of Children With Duchenne Muscular Dystrophy
This study aims to investigate the effects of urotherapy education provided to families of children with Duchenne muscular dystrophy on parents' knowledge, lifestyle changes, and satisfaction with the education.
- Trial name
- NCT07821125
- Testing
- Urotherapy Education
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Lokman Hekim University
Duchene Muscular Dystrophy
Whole-body Vibration Versus Gravity Force Stimulation on Postural Stability in Children With Duchene Muscular Dystrophy
this study aimed to mention Does whole-body vibration training or gravity-force stimulation produce greater improvements in postural stability among children with Duchenne Muscular Dystrophy?
- Trial name
- NCT07780435
- Testing
- force gravity stimulation, whole body vibration
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Cairo University
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Becker Muscular Dystrophy
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Monitoring patients with neuromuscular disorders is crucial but doesn't always allow for a sufficiently specific approach to all aspects of the condition.
- Trial name
- MyoBrain-BMD (NCT07740057)
- Where
- France
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
Nearly two-third of patients with myotonic dystrophy type 2 (DM2) report that impaired cognition is among the most disabling symptoms and deeply affects their quality of life.
- Trial name
- BraCE-DM2 (NCT05854433)
- Testing
- Non-interventional study
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Duchene Muscular Dystrophy, Becker Muscular Dystrophy
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in…
- Trial name
- INNOVAN (NCT07378553)
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Pediatric Radio Frequency Coils Generic
The purpose of this study is to evaluate and optimize advances in radio frequency (RF) coil magnetic resonance imaging (MRI) technology at Cincinnati Children's Hospital Medical Center (CCHMC).
- Trial name
- NCT01633866
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Duchenne Muscular Dystrophy (DMD)
A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin.
- Trial name
- TODDLER (NCT07423026)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Facio-Scapulo-Humeral Dystrophy
Computerized Facial Recognition for Automated Diagnosis of the Facio-Scapulo-Humeral Muscular Dystrophy (FSMHD)
The clinical diagnosis of Facio-Scapulo-Humeral Muscular Dystrophy (FSHMD) requires the movement of patients to a medical centre and a lengthy examination involving medical personnel, and may be underestimated in the most…
- Trial name
- CV4DIAGNOSIS (NCT04377217)
- Testing
- Video recording
- Where
- France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Nice
Duchenne Muscular Dystrophy
Wearable Technology to Evaluate Hyperglycemia and HRV in DMD
Duchenne muscular dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death.
- Trial name
- NCT06124196
- Testing
- wearable technology
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
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.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Neuromuscular Diseases
Muscle Oxygenation in Effort in Neuromuscular Diseases
Previous studies showed modifications of muscle oxygenation parameters in muscular dystrophies du to an impairment or an absence of dystrophin.
- Trial name
- OXYNEMU (NCT02789059)
- Testing
- muscle oxygenation
- Where
- 3 sites in France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University Hospital, Lille
FSH Muscular Dystrophy, Muscular Dystrophy
Amino Acids and Exercise in FSHD
This study evaluates whether amino acid supplementation, combined with a structured diet and exercise program, improves body composition (increased muscle mass and reduced fat mass) and physical performance in individuals with…
- Trial name
- NCT07543016
- Testing
- Essential Amino Acids Supplementation intervention, Exercise, Diet
- Where
- Italy
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- University of Pavia
Muscular Dystrophies, Congenital Myopathy
Assessment of Safety and Acute Effects of a Lower-limb Powered Dermoskeleton in Patients With Neuromuscular Disorders
The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a programmable ambulation exoskeleton (KeeogoTM Dermoskeleton System, B-Temia Inc., Quebec, Canada) in patients with…
- Conditions
- Muscular Dystrophies, Congenital Myopathy, Idiopathic Inflammatory Myopathies, Mitochondrial Myopathies
- Trial name
- Exo-KGO1 (NCT05199246)
- Testing
- Lower-limb powered dermoskeleton
- Where
- France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Muscular Dystrophies, Congenital Myopathy
Assessment of Safety and Acute Effects of a Knee-hip Powered Soft Exoskeleton in Patients With Neuromuscular Disorders
The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a powered knee-hip dermoskeleton (MyoSuit, MyoSwiss, Zurich, Switzerland) in patients with neuromuscular disorders, ii)…
- Conditions
- Muscular Dystrophies, Congenital Myopathy, Idiopathic Inflammatory Myopathies, Mitochondrial Myopathies
- Trial name
- Exo-NMD1 (NCT05200702)
- Testing
- Knee-hip Powered Soft Exoskeleton (MyoSuit, MyoSwiss, Zurich, Switzerland)
- Where
- France
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France