Clinical trials: Congenital Myopathy
Cleft Lip and Palate, Temporomandibular Disorder
Electromyographic Analysis of the Masticatory Muscles in Cleft Lip and Palate Children With Temporomandibular Disorders
The aim of this study was to assess the electrical activity of the temporal and masseter muscles in cleft lip and palate (CLP) children with pain-related temporomandibular disorders (TMD) and in CLP individuals with no TMD by…
- Trial name
- NCT03308266
- Testing
- electromyography
- Where
- Poland
- Volunteers
- 87 people
- Sponsor
- Pomeranian Medical University Szczecin
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
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Duchenne Muscular Dystrophy
Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
"Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy" is a single centre observational study that aims to follow the progress of 20 adolescents with Duchenne…
- Trial name
- NCT02571205
- Testing
- Sustanon (testosterone)
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
X Linked Myotubular Myopathy
Tamoxifen Therapy for Myotubular Myopathy
This is a phase 1 / 2, randomized, double-blinded, single cross-over study, with a washout period between treatment regimens, to test the efficacy and safety of tamoxifen therapy to improve motor and respiratory function in males…
- Trial name
- TAM4MTM (NCT04915846)
- Testing
- ApoTamox 10mg
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 6 people
- Sponsor
- James Dowling
Oculopharyngeal Muscular Dystrophy
Effect of Aerobic Training in Patients With Oculopharyngeal Muscular Dystrophy
The investigators want to investigate if patients with Oculopharyngeal Muscular Dystrophy can improve fitness by home-training on a cycle ergometer 30 minute, 3 times a week for 10 weeks.
- Trial name
- NCT02158156
- Testing
- Exercise
- Where
- Denmark
- Volunteers
- 8 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Duchenne Muscular Dystrophy
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT1-EDO51 (NCT06079736)
- Testing
- PGN-EDO51
- Where
- 5 sites in Canada
- Volunteers
- 7 people
- Sponsor
- PepGen Inc
Duchenne Muscular Dystrophy
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
- Trial name
- ENVOL (NCT06128564)
- Testing
- delandistrogene moxeparvovec
- Where
- 7 sites in Belgium, France and 4 more
- Volunteers
- 13 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Pediatric Radio Frequency Coils Generic
The purpose of this study is to evaluate and optimize advances in radio frequency (RF) coil magnetic resonance imaging (MRI) technology at Cincinnati Children's Hospital Medical Center (CCHMC).
- Trial name
- NCT01633866
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Incomplete Spinal Cord Injury, Muscular Dystrophy
Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders
Background: Cerebral palsy (CP) is the most common motor disorder in children. CP often causes crouch gait, an abnormal way of walking. Knee crouch has many causes, so no single device or approach works best for everybody.
- Conditions
- Incomplete Spinal Cord Injury, Muscular Dystrophy, Spina Bifida, Cerebral Palsy
- Trial name
- NCT01961557
- Testing
- EA-KAFO
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Congenital Myotonic Dystrophy
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
Cerebrovascular Accident, Post-polio Syndrome
Nomad P-KAFO Study
The goal of this clinical trial is to evaluate the impact of using the Nomad powered KAFO in people who have had a musculoskeletal or neurological injury that has affected their ability to walk.
- Conditions
- Cerebrovascular Accident, Post-polio Syndrome, Spinal Cord Injuries, Multiple Sclerosis
- Trial name
- NCT05644522
- Testing
- Indego Nomad® Powered Knee-Ankle-Foot Orthosis (P-KAFO) (Parker Hannifin Corp., Cleveland, OH)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Shirley Ryan AbilityLab
Muscular Dystrophy, Duchenne
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety…
- Trial name
- NCT02369731
- Where
- 71 sites in Austria, Brazil and 14 more
- Volunteers
- 316 people
- Sponsor
- PTC Therapeutics
Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI)
Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders
The primary aim of this observational study is to investigate specific aspects of social cognition in dystrophinopathies. Body awareness, interpersonal distance and emotional processing will be measured in a sample of patients…
- Conditions
- Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI), Social Cognition
- Trial name
- NCT06874166
- Testing
- Electroencephalography (EEG), Social Cognition Tasks
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Applying the Pathways and Resources for Engagement and Participation Protocol Among People With Muscles Dystrophies
Taking part in community activities is essential for health and well-being. Yet, it is highly restricted for young people with Duchenne and Becker Muscular Dystrophies (DBMD), especially as they grow into adulthood.
- Trial name
- NCT06540365
- Testing
- Pathways and Resources for Engagement and Participation (PREP)
- Volunteers
- 6 people
- Sponsor
- University of Haifa
Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Tolerability of Rituxan in Duchenne Muscular Dystrophy
1. Study population:It is applicable to male participants with genetically confirmed and clinically confirmed Duchenne muscular dystrophy (DMD), aged between 6 and 10 years. 2.
- Trial name
- NCT07475754
- Testing
- Rituxan treatment
- Volunteers
- 5 people
- Sponsor
- Peking Union Medical College Hospital
Muscular Dystrophies
First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect
The FTIH study with GSK3439171A will evaluate the safety of GSK3439171A in healthy subjects in order to avoid confounding factors due to the disease or concomitant drugs in patients.
- Trial name
- NCT03627494
- Testing
- GSK3439171A
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Muscular Dystrophy, Duchenne, Orthotic Devices
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.
- Conditions
- Muscular Dystrophy, Duchenne, Orthotic Devices, Upper Extremity, Neuromuscular Diseases (NMD)
- Trial name
- NCT06363357
- Testing
- Shoulder orthosis
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Seoul National University Hospital
Facioscapulohumeral Muscular Dystrophy, FSHD
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1
- Trial name
- FORTITUDE-3 (NCT07038200)
- Testing
- AOC-1020
- Where
- 46 sites in United States, Canada and 8 more
- Volunteers
- 200 people
- Sponsor
- Avidity Biosciences, Inc.
Mitochondrial Disorders, Mitochondrial Disease
Tissue Sample Study for Mitochondrial Disorders
The investigators are studying patients with undefined mitochondrial diseases to identify genetic mutations in nuclear or mitochondrial Deoxyribonucleic Acid (DNA).
- Conditions
- Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer
- Trial name
- NCT01803906
- Where
- United States
- Volunteers
- 6,900 people, healthy volunteers welcome
- Sponsor
- Columbia University
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.