Clinical trials: Muscular Dystrophy in Children
Duchenne Dystrophy Muscular
Biomechanical and Morphological Changes in Dystrophic Muscle
The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in…
- Trial name
- MARCHE-DMD (NCT02472990)
- Where
- 10 sites in France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
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
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
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
Telerehabilitation-based Motor Imagery Training in Children With Duchenne Muscular Dystrophy
When the field of neurorehabilitation is examined, most of the current physiotherapy and rehabilitation approaches are based on real movements to stimulate damaged motor neural connections through neuroplasticity.
- Trial name
- NCT06109103
- Testing
- telerehabilitation-based motor imagery training, telerehabilitation-based physiotheraphy training
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
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
Duchene Muscular Dystrophy
How Can Different Forms of Strengthening Exercises Affect the Muscular Tissue and Its Chemical Markers in Children With Duchenne Muscular Dystrophy
The treatment of patients diagnosed with DMD needs to be multidisciplinary, judicious, and always focused on the patient's well-being. Physiotherapy and occupational therapy are very often standard care.
- Trial name
- NCT07581743
- Testing
- strengthening exercises, Low-Intensity Exercise Protocol, traditional physical therapy program
- Where
- Egypt
- Volunteers
- 45 people
- Sponsor
- Cairo University
Muscular Dystrophy, Duchenne
Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy
This study was planned to investigate the parental influence on physical activity (PA) level and participation in ambulatory children with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05209087
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
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, 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
Aerobic Exercise in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease in childhood with an estimate incidence of 1 in 3500 to 5000 male births.
- Trial name
- NCT04173234
- Testing
- Aerobic Training, Home exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Hacettepe University
Becker Muscular Dystrophy, Muscular Dystrophies
Defining Endpoints in Becker Muscular Dystrophy
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Trial name
- GRASP-01-002 (NCT05257473)
- Where
- 11 sites in United States, New Zealand and 1 more
- Volunteers
- 59 people
- Sponsor
- Virginia Commonwealth University
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
The goal of this observational study is to investigate whether the type, location, and extent of pathogenic variants in the DMD gene are associated with cardiac dysfunction in male children, adolescents, and young adults with…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Cardiomyopathy
- Trial name
- NCT07515235
- Where
- Greece
- Volunteers
- 65 people
- Sponsor
- Aristotle University Of Thessaloniki
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
Duchenne Muscular Dystrophy, Muscle Strength
Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy
Investigator researched that the effect of trunk and lower extremity muscle strength on hamstring flexibility in children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Muscle Strength, Lower Extremity, Hamstring Contractures
- Trial name
- NCT03589573
- Where
- Turkey (Türkiye)
- Volunteers
- 30 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
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
Upper Extremity Muscle Strength, Balance and Functional Skills in DMD
This study aims to examine the relationship between upper extremity muscle strength, balance and functional skills of children with DMD.
- Trial name
- NCT06379906
- Testing
- Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Halic University
Duchenne Muscular Dystrophy
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD).
- Trial name
- IGNITE DMD (NCT03368742)
- Testing
- SGT-001
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Solid Biosciences Inc.
Duchenne Muscular Dystrophy
Motor Imagery on Children With DMD on Gait and Balance Functions
The most common muscular dystrophy among pediatric neuromuscular diseases is Duchenne Muscular Dystrophy (DMD). There is no consensus on a standardized physiotherapy and rehabilitation program or exercise prescription in DMD.
- Trial name
- NCT05601986
- Testing
- Physiotherapy and Rehabilitation Program, Additional Motor Imagery Training to Physiotherapy and Rehabilitation Program
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people
- Sponsor
- Gülsena Utku