Clinical trials: Congenital Myopathy
Duchenne Muscular Dystrophy
Stacking Exercises Aid the Decline in FVC and Sick Time
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths…
- Trial name
- STEADFAST (NCT01999075)
- Testing
- Lung Volume Recruitment (LVR), Conventional Treatment
- Where
- 9 sites in Canada
- Volunteers
- 70 people
- Sponsor
- Children's Hospital of Eastern Ontario
Nemaline Myopathy
Treatment of TNNT1-Myopathy With L-Tyrosine.
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.
- Trial name
- NCT02035501
- Testing
- L-Tyrosine
- Where
- Israel
- Volunteers
- 10 people
- Sponsor
- Hadassah Medical Organization
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a…
- Trial name
- NCT02819557
- Testing
- Ataluren
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory…
- Trial name
- HORIZON (NCT06597656)
- Testing
- delandistrogene moxeparvovec, Plasmapheresis
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
The Effect of Dual-tasking Program on Cognitive and Physical Functions and Independence in Activities of Daily Living in Children With Duchenne Muscular Dystrophy: A Single-blind Randomized Controlled Trial
Duchenne Muscular Dystrophy (DMD) is an X-linked recessive disorder characterized by progressive muscle degeneration and frequent developmental, cognitive and behavioral impairments, occurring in one in 5000 live births of boys.
- Trial name
- DMD-DUAL-FUNC (NCT06887491)
- Testing
- Conventional physiotherapy program, Conventional physiotherapy program + Dual task program
- Where
- Turkey (Türkiye)
- Volunteers
- 16 people
- Sponsor
- Lokman Hekim University
Bethlem Myopathy, Ullrich Congenital Muscular Dystrophy
Low Protein Diet in Patients With Collagen VI Related Myopathies
This is a 2 stage exploratory study with a 3-month observational phase on the natural course, followed by a 12-month, open-label, non-comparative, single-arm, phase II pilot study on the efficacy, safety and tolerability of a…
- Trial name
- LPD (NCT01438788)
- Testing
- Low protein diet
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Istituto Ortopedico Rizzoli
Duchenne Muscular Dystrophy
Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy. II.
- Trial name
- NCT00004646
- Testing
- prednisone
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)
Duchenne Muscular Dystrophy (DMD), Lower Urinary Track Symptoms
Effects of Lumbopelvic Stabilization-based Physiotherapy and Rehabilitation Training in Duchenne Muscular Dystrophy
This study aims to examine the effectiveness of supervised lumbopelvic stabilization in relation to factors associated with lower urinary tract symptoms in children diagnosed with Duchenne Muscular Dystrophy (DMD) who have lower…
- Trial name
- NCT06643923
- Testing
- Exercise, Urotherapy
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people
- Sponsor
- Lokman Hekim University
Duchenne Muscular Dystrophy (DMD)
Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
The aim of this study is to verify whether full video home polysomnography (sleep study) with nocturnal transcutaneous CO2 monitoring is feasible in the follow-up of non-invasive ventilation (NIV) in patients with Duchenne…
- Trial name
- NCT06773988
- Testing
- Monitoring of NIV at home
- Where
- Belgium
- Volunteers
- 17 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Duchenne Muscular Dystrophy (DMD)
DMD Voice: Qualitative Interviews With Patients and Caregivers
The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers.
- Trial name
- NCT06925269
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Red Nucleus Enterprise Solutions, LLC
Myotonic Muscular Dystrophy
Study of Muscle Wasting and Altered Metabolism in Patients With Myotonic Dystrophy
OBJECTIVES: I. Examine the interrelationships between muscle wasting (phenotype), the degree of myotonic dystrophy (DM) gene expression (genotype) in patients with DM. II. Characterize the insulin resistance in these patients.
- Trial name
- NCT00004769
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Duchenne Muscular Dystrophy
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02858362
- Testing
- Ezutromid
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 43 people
- Sponsor
- Summit Therapeutics
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of…
- Trial name
- NCT03439670
- Testing
- Vamorolone, Prednisone
- Where
- 33 sites in United States, Australia and 9 more
- Volunteers
- 121 people
- Sponsor
- ReveraGen BioPharma, Inc.
Congenital Myopathy, Acquired Myopathy
Magnetic Resonance Imaging and Ultrasonography in Evaluation of Muscle Diseases
This study aims to determine the association between the pathological changes detected by ultrasound and those detected in MRI in muscle diseases of different etiologies.
- Conditions
- Congenital Myopathy, Acquired Myopathy, Myositis
- Trial name
- NCT05692349
- Testing
- MRI
- Volunteers
- 36 people
- Sponsor
- Assiut University
Spina Bifida, Neuromuscular Diseases
Metformin in Children With Motor Deficit
Obesity with insulin resistance in the paediatric population provides an increasing challenge. Children with neurological or neuromuscular diseases are even more prone to obesity: their locomotor impairment leads to an…
- Trial name
- NCT00720161
- Testing
- Metformin
- Where
- Belgium
- Volunteers
- 42 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Duchenne Muscular Dystrophy
Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
This study, supported by Parent Project Muscular Dystrophy, will determine if tadalafil or sildenafil can improve muscle blood flow during exercise in boys with Duchenne muscular dystrophy.
- Trial name
- NCT01359670
- Testing
- Tadalafil, Sildenafil
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Cedars-Sinai Medical Center
Congenital Myotonic Dystrophy
Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy
Congenital Myotonic Dystrophy (CDM) is a multi-systemic, dominantly inherited disorder caused by a trinucleotide repeat expansion (CTGn) in the DMPK gene.
- Trial name
- TREAT-CDM (NCT03059264)
- Testing
- Natural history
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Virginia Commonwealth University
Duchenne Muscular Dystrophy
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases…
- Trial name
- FOR-DMD (NCT01603407)
- Testing
- Prednisone, Deflazacort
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 196 people
- Sponsor
- University of Rochester
Myotonic Dystrophy 1
Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
Impairment of balance and gait are frequent complaints in patients with myotonic dystrophy type 1 (DM1). In these persons, there is an increased risk for stumbles and falls when compared to normal subjects.
- Trial name
- CABLAMYD (NCT04712422)
- Testing
- Healthy subjects, Pathologic group
- Where
- 2 sites in Italy
- Volunteers
- 42 people
- Sponsor
- Istituto Auxologico Italiano
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.