Clinical trials: Muscular Dystrophy in Children
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, 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
Congenital Myotonic Dystrophy
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
- Trial name
- NCT03692312
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 56 people
- Sponsor
- AMO Pharma Limited
Duchenne Muscular Dystrophy (DMD)
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
The aim of the BABY DUCHENNE study is to evaluate the natural history and characterize the early clinical outcomes in very young children (0-3 years) with Duchenne muscular dystrophy (DMD) identified by newborn screening programs.
- Trial name
- BABY DUCHENNE (NCT07092540)
- Where
- 4 sites in United States
- Volunteers
- 105 people
- Sponsor
- University of Rochester
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
Duchenne Muscular Dystrophy
2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children
Compare systolic function of left ventricle (LV) and right ventricle (VD) by 2D strain evaluation in Duchenne muscular dystrophy children versus a control group.
- Trial name
- NCT02418338
- Testing
- speckle tracking (2D strain) echocardiography
- Where
- France
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Cardiac Outcome Measures in Children With Muscular Dystrophy
The purpose of the research study is to evaluate different cardiac measures that are obtained by echocardiographic tests in patients with muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT01066455
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Cooperative International Neuromuscular Research Group
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
Muscular Dystrophy, Becker, Muscular Dystrophy
A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children
This is a prospective, multicenter, longitudinal observational cohort study aimed at understanding the progression of Duchenne Muscular Dystrophy (DMD).
- Conditions
- Muscular Dystrophy, Becker, Muscular Dystrophy, Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
- Trial name
- WEST-DBMD (NCT07511920)
- Testing
- No interventions are applied; participants are followed for natural history observation only
- Where
- China
- Volunteers
- 500 people
- Sponsor
- West China Second University Hospital
Neuromuscular Diseases in Children, Respiratory Function Impaired
Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
The clinical trial titled "Investigation of Respiratory Functions, Thoracoabdominal Movements, and Exercise Capacity in Neuromuscular Diseases" aims to evaluate the respiratory functions, thoracoabdominal movements, and exercise…
- Conditions
- Neuromuscular Diseases in Children, Respiratory Function Impaired, Respiratory Insufficiency, Duchenne Muscular Dystrophy
- Trial name
- NCT06756633
- Testing
- Respiratory Function Test, Structured light plethysmography, 6 Minute Walking Test
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Lokman Hekim University
Duchenne Muscular Dystrophy
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
- Trial name
- NCT02251600
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Duchenne's Muscular Dystrophy (DMD)
L-citrulline and Metformin in Duchenne's Muscular Dystrophy
The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.
- Trial name
- NCT01995032
- Testing
- 750 mg metformin and 7.5 g L-citrulline daily p.o.
- Where
- Switzerland
- Volunteers
- 47 people
- Sponsor
- University Hospital, Basel, Switzerland
Neurological Observations, Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Dystrophy in Children
This study will focus on the symptoms, natural history and clinical impact of facioscapulohumeral muscular dystrophy (FSHD) in children. Symptoms of classical FSHD start in adulthood.
- Conditions
- Neurological Observations, Facioscapulohumeral Muscular Dystrophy, Pediatric Disorder
- Trial name
- iFocus (NCT02625662)
- Where
- Netherlands
- Volunteers
- 32 people
- Sponsor
- University Medical Center Nijmegen
Duchenne Muscular Dystrophy (DMD)
Peabody Developmental Motor Scaling In Children With Period Duchenne Muscular Dystrophy
To perform the reliability and validity study of the Peabody Developmental Motor Scale (Peabody Developmental Motor Scales-2) in children with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT05990608
- Testing
- Peabody Developmental Motor Scales-2, PDMS
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ondokuz Mayıs University
Inflammatory Myopathy, Duchenne Muscular Dystrophy
Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
The aim of our study is to Assess skeletal muscle structural status in children with inflammatory myositis and Duchenne muscular dystrophy using musculoskeletal ultrasound and to perform a longitudinal follow up of these changes…
- Trial name
- NCT03786913
- Testing
- Quantitative muscle ultrasound measurements
- Where
- Egypt
- Volunteers
- 48 people
- Sponsor
- Benha University
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
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
Muscular Dystrophy
Heart Imaging in Children With Muscular Dystrophy
Background: Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function.
- Trial name
- NCT01633242
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Duchenne Muscular Dystrophy (DMD)
Virtual Reality and Whole Body Vibration on Muscle Strength and Balance in Children With DMD
we measure balance and quadriceps strength in Duchenne muscular dystrophy children after applying virtual reality and whole body vibration
- Trial name
- NCT07338812
- Testing
- virtual reality, whole body vibration
- Where
- 2 sites in Egypt
- Volunteers
- 50 people
- Sponsor
- Cairo University
Congenital Muscular Dystrophy
Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil. Funding source - FDA OOPD
- Trial name
- CALLISTO (NCT01805024)
- Testing
- Omigapil
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Santhera Pharmaceuticals