Clinical trials: Muscular Dystrophy in Children
Duchenne Muscular Dystrophy, Pompe Disease (Late-onset)
Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease
This is a multicenter prospective non-drug screening study. The working period is 12 months. There is no research product to be followed or used in the study.
- Trial name
- VICTORIA (NCT04120168)
- Testing
- Laboratory Tests
- Where
- 51 sites in Turkey (Türkiye)
- Volunteers
- 590 people
- Sponsor
- Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition
Facio-Scapulo-Humeral Dystrophy, FSHD2
Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
This multi-centre, randomised, double-blind, placebo-controlled crossover trial will compare changes in strength-related motor function following treatment with creatine monohydrate to treatment with placebo, as measured by the…
- Conditions
- Facio-Scapulo-Humeral Dystrophy, FSHD2, FSHD1
- Trial name
- NCT02948244
- Testing
- Creatine Monohydrate
- Where
- Australia
- Volunteers
- 11 people
- Sponsor
- Murdoch Childrens Research Institute
Duchene's Muscular Dystrophy
Analysis of Motor Performance in Children with Duchene's Muscular Dystrophy
25 boys, ages ranging from 6 to 16 years, diagnosed with Duchene's muscular Dystrophy (DMD) will participated in the study. They will be selected from the Pediatric Rehabilitation Center of Deraya University's outpatient clinic…
- Trial name
- NCT06750042
- Volunteers
- 25 people
- Sponsor
- Cairo University
Duchenne Muscular Dystrophy
Dual Task in Duchenne Muscular Dystrophy
This study was planned to determine the effects of the dual-task performance of children with DMD with motor dysfunction and varying degrees of cognitive impairment compared to their healthy peers, to compare the dual-task…
- Trial name
- NCT05575648
- Testing
- 10 meter walk test
- Where
- Turkey (Türkiye)
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy, Duchenne Disease
Vasodilator and Exercise Study for DMD (VASO-REx)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved…
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children
- Trial name
- NCT06290713
- Testing
- Tadalafil, Exercise Training
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Florida
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
DMD-associated Dilated Cardiomyopathy
Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
This study aims at exploring the use of empagliflozin in children and adolescents 6-18 years old with Duchenne muscular distrophy (DMD) - associated cardiomyopathy.
- Trial name
- REDMeD (NCT06643442)
- Testing
- Empagliflozin Tablets
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Sebastiano Lava
Duchenne Muscular Dystrophy, Duchenne Disease
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
This pilot study will investigate the potential efficacy of VM100, a nutritional supplement specifically formulated for patients with DMD, on quality of life and physical symptoms.
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD
- Trial name
- NCT07766980
- Testing
- VM100 Nutritional Supplement
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Florida
Muscular Dystrophies, Congenital Myopathy
Bullying in Youth With Muscular Dystrophy and Congenital Myopathies
Bullying is an epidemic in Canada, and rates may be underreported. Youth with a disability were more likely to be bullied that those without disabilities, specifically if the disability was visible.
- Trial name
- NCT04733976
- Where
- 2 sites in Canada
- Volunteers
- 29 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Duchenne Muscular Dystrophy, Gait Disorders in Children
Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD)
The aim of this study was to develop a reliable and valid gait classification scale for Duchenne Muscular Dystrophy (QIGS-DMD). The items of the QIGS-DMD were designed based on the literature review considering existing…
- Trial name
- NCT05564962
- Testing
- Quality and Independence of Gait Classification Scale for Duchenne Muscular Dystrophy (QIGS-DMD)
- Where
- Turkey (Türkiye)
- Volunteers
- 69 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies.
The purpose of this study is to analyze the effectiveness of a 5-weeks respiratory digital intervention program in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Muscular Dystrophy
- Trial name
- DMDrespy2024 (NCT06363526)
- Testing
- Digital respiratory intervention program
- Where
- Spain
- Volunteers
- 12 people
- Sponsor
- University of Malaga
Duchenne Muscular Dystrophy
The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy
Children with Duchenne Muscular Dystrophy (DMD) have difficulties towards the end of the ambulatory period, especially in activities that require lower extremity proximal muscle strength such as walking, climbing stairs, standing…
- Trial name
- NCT04287582
- Testing
- Electromyographic device
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
This is an investigation of the efficacy and safety of CRD007 in Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers.
- Trial name
- NCT01540604
- Testing
- CRD007
- Where
- Sweden
- Sponsor
- RSPR Pharma AB
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
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
Physical Activity Level and Cognitive Functions in Children With Duchenne Muscular Dystrophy
Although there are studies showing that the effect on motor performance over time in children with DMD is associated with a decrease in the level of physical activity, no publication has been found that directly examines the…
- Trial name
- NCT05641805
- Testing
- PAQ-C (Physical activitiy questionare for children), MoCA (Montreal Cognitive Assessment), PBS (Pediatric Balance Scale), Modified Mini Mental State Examination, BMI (Body Mass Index)
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Muş Alparslan University
Duchenne Muscular Dystrophy
Effect of Kinesiology Taping on Head and Trunk Control in Children With Duchenne Muscular Dystrophy
Muscular dystrophy (MD) is a group of muscle diseases that results in increasing weakening and breakdown of skeletal muscles over time.
- Trial name
- NCT05967793
- Testing
- kinesio tape
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Egyptian Chinese University
Developmental Disability, Intellectual Disability
Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort
The purpose of this research use only (RUO) study is to detect genomic structural variants (SVs) in human DNA by Optical Genome Mapping (OGM) using the Bionano Genomics Saphyr system.
- Conditions
- Developmental Disability, Intellectual Disability, Autism Spectrum Disorder, Congenital Anomaly
- Trial name
- NCT05295277
- Testing
- Standard of care genetic testing group
- Where
- 8 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Bionano Genomics
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
Increase Muscle Strength in Patients With Muscular Dystrophy
Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
Importance: Currently the gold standard treatment for ambulant patients is corticosteroids. Granulocyte colony-stimulating factor (G-CSF) has been reported to exert the proliferation of satellite cells, the regulation of myoblast…
- Trial name
- NCT02814110
- Testing
- Granulocyte colony-stimulating factor (Filgrastim)
- Where
- Poland
- Volunteers
- 27 people
- Sponsor
- Medical University of Bialystok