Clinical trials: Congenital Myopathy
Limb Girdle Muscular Dystrophy
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- NCT05876780
- Testing
- SRP-9003
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Exploratory Study of NS-089/NCNP-02 in DMD
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies.
- Trial name
- NCT04129294
- Testing
- NS-089/NCNP-02
- Where
- Japan
- Volunteers
- 6 people
- Sponsor
- National Center of Neurology and Psychiatry, Japan
Duchenne Muscular Dystrophy
Bicycle Training on Ventilatory Functions in Duchenne Muscular Dystrophy
The purpose of the study is to investigate the effect of bicycle ergometry training on ventilatory functions in boys with Duchenne muscular dystrophy.
- Trial name
- NCT05849688
- Testing
- Designed Physical Therapy Program, Bicycle Ergometry Training
- Volunteers
- 30 people
- Sponsor
- Beni-Suef University
Duchenne Muscular Dystrophy
Natural History of Duchenne Muscular Dystrophy
Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate…
- Trial name
- NCT03882827
- Where
- 15 sites in Belgium, France and 2 more
- Volunteers
- 220 people
- Sponsor
- Genethon
Limb-Girdle Muscular Dystrophy, Type 2E
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
This study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial.
- Trial name
- NCT03492346
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Nationwide Children's Hospital
Inflammation, Spinal Cord Injuries
Anti-inflammatory Diet Consultation for Those With Neuromuscular Disability
This study investigated the effects of a 2-part dietary consultation on adherence to an anti-inflammatory diet in individuals with neuromuscular disability.
- Conditions
- Inflammation, Spinal Cord Injuries, Multiple Sclerosis, Muscular Dystrophies
- Trial name
- NCT05881122
- Testing
- Mad Dog Dietary Consultation
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- Brock University
Choreatic Disorder
Heart and Skeletal Muscle Problems in Neuroacanthocytosis
The purpose of this study is to learn about heart and skeletal muscle problems related to neuroacanthocytosis (also called Mcleod's syndrome and Levine-Critchley disease).
- Trial name
- NCT00007228
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and < 7 years old.
- Trial name
- NCT02760264
- Testing
- Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
TMJ Disorder, Mandibular Deficiency
Virtual Planning of Total TMJ Replacement
Virtual Planning of Total TMJ Replacement
- Conditions
- TMJ Disorder, Mandibular Deficiency, TMJ Ankylosis
- Trial name
- TJR (NCT07643259)
- Testing
- Custom made TMJ
- Where
- Egypt
- Volunteers
- 8 people
- Sponsor
- Faculty of Dental Medicine for Girls
Muscular Dystrophies
Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)
This is a study to determine whether albuterol or oxandrolone, alone or in combination, are able to increase strength and muscle mass in patients with FSHD.
- Trial name
- NCT00027391
- Testing
- Albuterol, Oxandrolone
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- FDA Office of Orphan Products Development
Duchenne Muscular Dystrophy
Phase I/II Study of SRP-4053 in DMD Patients
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
- Trial name
- NCT02310906
- Testing
- SRP-4053
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Sarepta Therapeutics, Inc.
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
The investigators are performing a gene therapy clinical trial in Becker muscular dystrophy (BMD) and sporadic inclusion body myositis (sIBM) patients.
- Trial name
- NCT01519349
- Testing
- rAAV1.CMV.huFollistatin344
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy
The Effect of Telerehabilitation of Patients With Duchenne Muscular Dystrophy
The aim of this study is to investigate the effect of a telerehabilitation approach to patients with Duchenne Muscular Dystrophy and evaluate patients' motor function, parents' anxiety and depression levels before and after the…
- Trial name
- NCT04782440
- Testing
- Telerehabilitaton, Home exercise
- Volunteers
- 20 people
- Sponsor
- Marmara University
Limb Girdle Muscular Dystrophy, Muscular Dystrophies
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles.
- Trial name
- GRASP-01-001 (NCT03981289)
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 116 people
- Sponsor
- Virginia Commonwealth University
Facio-Scapulo-Humeral Dystrophy
FSHD Molecular Characterization
To characterize the clinical and molecular phenotype of FSHD.
- Trial name
- NCT06096441
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Nationwide Children's Hospital
Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy
UTSW HP [13-C] Pyruvate Injection in HCM
The study objective is to identify the earliest changes in energy substrate metabolism in patients with cardiomyopathies (CMP). To achieve this objective, we plan first to test the hypothesis that patients with CMP present focal…
- Conditions
- Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis
- Trial name
- HPHCM (NCT03057002)
- Testing
- Hyperpolarized 13C-Pyruvate
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Duchenne Muscular Dystrophy, Exon 44
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping.
- Conditions
- Duchenne Muscular Dystrophy, Exon 44, DMD
- Trial name
- NCT05996003
- Testing
- NS-089/NCNP-02
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Males With X-linked Myotubular Myopathy (XLMTM)
A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)
This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal to obtain 50 deceased and 20 living records) will provide further knowledge about the clinical manifestations and recorded…
- Trial name
- NCT02231697
- Testing
- Non-interventional, retrospective medical chart review
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 161 people
- Sponsor
- Astellas Gene Therapies
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]
Until twenty years ago physical exercise in muscular dystrophies was considered harmful to the muscle cells, inducing an acceleration of cell necrosis.
- Conditions
- Muscular Dystrophies, Limb Girdle Muscular Dystrophy, Facio-Scapulo-Humeral Dystrophy, Becker Muscular Dystrophy
- Trial name
- RIMUDI (NCT06378203)
- Testing
- tele-rehabilitation system with virtual reality Home Kit - Virtual Reality Rehabilitation System (VRRS KHYMEIA)
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS Eugenio Medea
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