Clinical trials: Muscular Dystrophy in Children
Duchenne Muscular Dystrophy, Lower Urinary Tract Symptoms
Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy
The aim of this study is to examine the prevalence of lower urinary tract symptoms (LUTS) in children with Duchenne Muscular Dystrophy (DMD) and the relationship between functional level, posture, muscle strength, pelvic floor…
- Conditions
- Duchenne Muscular Dystrophy, Lower Urinary Tract Symptoms, Muscle Weakness, Posture Disorders in Children
- Trial name
- NCT05464446
- Testing
- Baseline Bubble Inclinometer, Baseline Digital İnclinometer and Hoggan microFET2 devices
- Where
- Turkey (Türkiye)
- Volunteers
- 45 people
- Sponsor
- Lokman Hekim University
Duchenne Muscular Dystrophy, Upper Limb Function
Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy
Although it is known that the functions of pulmonary and upper limb is affected in late stage of Duchenne Muscular Dystrophy (DMD) negatively, the investigators do not have clear information about its early stage.
- Conditions
- Duchenne Muscular Dystrophy, Upper Limb Function, Respiratory Function Test
- Trial name
- NCT03552874
- Testing
- The Performance of Upper Limb Questionnaire (PUL)
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy (DMD), Neuromuscular Disorders
Relation Between Muscle Architecture and Functional Ability in Children With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive X-linked neuromuscular disorder characterized by muscle degeneration, pseudohypertrophy, and declining functional mobility.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Neuromuscular Disorders, Muscle Architecture
- Trial name
- NCT07437378
- Where
- Egypt
- Volunteers
- 26 people
- Sponsor
- Deraya University
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
Duchenne Muscular Dystrophy
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized…
- Trial name
- FOX (NCT06100887)
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 7 sites in United States
- Volunteers
- 43 people
- Sponsor
- Edgewise Therapeutics, Inc.
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy (DMD), Chronic Pain
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
1. Brief Summary This prospective observational study aims to evaluate the 6-month longitudinal changes in chronic pain, pain characteristics, coping strategies, and functional performance in children and adolescents aged 7 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Chronic Pain, Neuromuscular Diseases (NMD)
- Trial name
- NCT07892196
- Testing
- Routine Clinical Follow-up and Pain Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Antalya Training and Research Hospital
Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
- Trial name
- NCT02295748
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.
Duchenne Muscular Dystrophy
Motor Imagery in Duchenne Muscular Dystrophy
Motor imagery (MI) can be defined as a dynamic process in which the person is mentally stimulated without performing the given motor movement.
- Trial name
- NCT05559710
- Where
- Turkey (Türkiye)
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Hacettepe University