Clinical trials: Muscular Dystrophy
Bethlem Myopathy
The Effect of Aerobic Exercise, on Fitness and Functional Muscle Strength, in Patients With Muscular Dystrophy
Muscular dystrophy is a group of disorders that are characterized by progressive muscle weakening and loss of muscle mass, caused by defects in muscle proteins.
- Trial name
- NCT01895283
- Testing
- Moderate-intensity training
- Where
- Denmark
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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
Duchenne Muscular Dystrophy, Gait Disorders in Children
Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy
The aim of this study was to develop a gait assessment instrument for Duchenne Muscular Dystrophy patients (DMD-GAS), and investigate its validity and reliability.The scale was developed considering the expert opinions which…
- Conditions
- Duchenne Muscular Dystrophy, Gait Disorders in Children, Gait Disorders, Neurologic
- Trial name
- NCT05244395
- Testing
- Duchenne Muscular Dystrophy Gait Assessment Scale
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy
This study is to determine whether physiologic measures (peak cough flow, measures of respiratory muscle strength including MIP, MEP ,SNIP, and spirometry) can predict spontaneous cough clearance (as measured by a nuclear…
- Trial name
- NCT02034305
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of Pittsburgh
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Duchenne Muscular Dystrophy
Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo…
- Trial name
- NCT04740554
- Testing
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone, Duchenne Muscular Dystrophy group without Corticosteroids therapy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Muscular Dystrophies, Muscle Inflammation
Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies
The study aimed to assess the safety and, partially, the efficacy of dietary supplementation of a flavonoids-, DHA- and EPA-based natural supplement in non-ambulant DMD boys and in a cohort of LGMD and FSHD patients to compare…
- Trial name
- PRO1 (NCT03317171)
- Testing
- Flavonoids, DHA, EPA
- Where
- Italy
- Volunteers
- 29 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Muscular Dystrophy, Duchenne Type
Trunk Oriented Exercises Versus Whole-body Vibration for Duchenne Muscular Dystrophy
The abdominal muscles play an important role in stabilizing the trunk and providing postural stability. Children with Duchene muscular dystrophy have weak muscles, which may impair postural adjustments.
- Trial name
- NCT05688072
- Testing
- trunk oriented exercise, whole body vibration
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Cairo University
LGMD2I, LGMDR9
Global FKRP Registry
Mutations in the Fukutin Related Protein (FKRP) gene cause the condition Limb Girdle Muscular Dystrophy type R9 (LGMDR9) also known as LGMD2I, and the rarer conditions Congenital Muscular Dystrophy (MDC1C), Muscle Eye Brain…
- Conditions
- LGMD2I, LGMDR9, Limb Girdle Muscular Dystrophy, Congenital Muscular Dystrophy
- Trial name
- NCT04001595
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Newcastle University
Duchenne Muscular Dystrophy, Performance
Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy
Investigator investigated that regression of hamstring flexibility and performance in children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Performance, Flexibility
- Trial name
- NCT03589612
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment…
- Trial name
- ENDURE (NCT06270719)
- Testing
- Delandistrogene Moxeparvovec, Standard of Care
- Where
- 29 sites in United States
- Volunteers
- 500 people
- Sponsor
- Sarepta Therapeutics, Inc.
Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD)
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy…
- Trial name
- Active-NBS BE (NCT07286565)
- Testing
- MAIJU, Syde, Questionnaires
- Where
- Belgium
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems.
- Trial name
- NCT06503367
- Where
- 14 sites in United States
- Volunteers
- 44 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2)…
- Trial name
- MOMENTUM (NCT04004065)
- Testing
- Vesleteplirsen
- Where
- 25 sites in United States, Belgium and 6 more
- Volunteers
- 62 people
- Sponsor
- Sarepta Therapeutics, Inc.
Congenital Myopathy, Neuromuscular Diseases
Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment
Congenital myopathies (CM) is a large group of muscle disorders, presenting with hypotonia and non-progressive generalised muscle weakness, which can lead to motor developmental delay.More than 20 genes can cause CM and currently…
- Conditions
- Congenital Myopathy, Neuromuscular Diseases, Musculoskeletal Diseases, Nemaline Myopathy
- Trial name
- COMPIS (NCT05099107)
- Testing
- Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet, Salbutamol Only Product in Oral Dose Form
- Where
- Sweden
- Volunteers
- 18 people
- Sponsor
- Vastra Gotaland Region
Muscular Dystrophy, Facioscapulohumeral
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups.
- Trial name
- NCT06517498
- Testing
- No Intervention
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Xi'an Jiaotong University
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Myotonic Dystrophy
Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy
Myotonic dystrophy (dystrophia myotonica; DM), the most prevalent form of muscular dystrophy in adults, is characterized by progressive myopathy, myotonia, and multi-systemic involvement.
- Trial name
- DeQoDE-DM (NCT07362875)
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences