Clinical trials: Muscular Atrophy
Sarcopenia, Juvenile Idiopathic Arthritis
Sarcopenia in Children
Juvenile idiopathic arthritis (AJI), is a frequent inflammatory disease in children, characterized by pain, arthritis, and deformities.
- Trial name
- NCT05291416
- Testing
- densitometry/ handgrip dynamometer
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Tunis El Manar
Congenital Myopathies, Muscular Dystrophy
Personalized Disease Modeling for Rare Genetic Myopathies
The goal of this observational study is to learn how rare genetic muscle diseases change over time and whether muscle cells grown from a participant's blood can help researchers understand the disease.
- Trial name
- NCT07914049
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Akron Children's Hospital
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.
Muscular Dystrophy, Facioscapulohumeral
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
- Trial name
- MOVE Peds (NCT06847282)
- Where
- 7 sites in United States, Australia
- Volunteers
- 80 people
- Sponsor
- University of Kansas Medical Center
Spinal Muscular Atrophy Type I
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy type I; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE 02 (NCT00439218)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Westat
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-45 (NCT07038824)
- Testing
- ENTR-601-45
- Where
- 15 sites in Belgium, Italy and 3 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Duchenne Muscular Dystrophy
Virtual Reality in Individuals With Duchenne Muscular Dystrophy
With the growing accessibility of computer-assisted technology, one option for rehabilitation programs for individuals with Duchenne muscular dystrophy (DMD) is the use of virtual reality environments to enhance motor practice.
- Trial name
- NCT03002298
- Testing
- DMD gesture task, DMD button-press task, Control group gesture task
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Progeria
Clinical Studies of Progeria
This study will examine children with Hutchinson-Gilford Progeria syndrome, a genetic disease that causes many changes to the body over time, including heart disease, bone changes, hair loss, and joint and skin changes.
- Trial name
- NCT00094393
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Hip Fractures, Sarcopenia
Feasibility of High Density Sit-to-stand Functional Resistance Training in Patients With Hip Fracture
The objective of this study is to examine if functional high-density, high-volume chair rise training is feasible for increasing lower extremity strength without causing excessive hip pain during execution in patients with hip…
- Conditions
- Hip Fractures, Sarcopenia, Fall Injury
- Trial name
- NCT05840315
- Testing
- Basic intervention and nutritional intervention, Sit-to-stand, Leg press
- Where
- Denmark
- Volunteers
- 30 people
- Sponsor
- Horsholm Municipality
Muscular Atrophy, Spinal
A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam.
- Trial name
- NCT07378943
- Where
- United States
- Volunteers
- 4,114 people
- Sponsor
- Novartis Pharmaceuticals
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
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.
- Trial name
- NCT01826474
- Testing
- PRO045, 0.15 mg/kg/week, PRO045, 1.0 mg/kg/week, PRO045, 3.0 mg/kg/week
- Where
- 6 sites in Belgium, France and 3 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
- Trial name
- NCT04687020
- Testing
- Viltolarsen
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- NS Pharma, Inc.
Myotonic Dystrophy Type 1, DM1
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia
- Trial name
- HARBOR-OLE (NCT07008469)
- Testing
- Del-desiran (AOC 1001)
- Where
- 35 sites in United States, Canada and 8 more
- Volunteers
- 199 people
- Sponsor
- Avidity Biosciences, Inc.
Muscular Diseases, Spinal Muscular Atrophy
Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)
This study aims to refine the capability of Multispectral Optoacoustic Tomography (MSOT) and Magnet Resonance Imaging (MRI) to characterise the molecular composition of muscle tissue non-invasively and to evaluate the therapeutic…
- Trial name
- NCT04262570
- Testing
- Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- University of Erlangen-Nürnberg Medical School
SMA - Spinal Muscular Atrophy
An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)
An Open Label Individual Patient (OLIP) Study of Nusinersen (BIIB058) in a Participant with Spinal Muscular Atrophy Who Previously Participated in an Investigational Study with Nusinersen
- Trial name
- NCT07846696
- Testing
- Nusinersen Injectable Product
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Spinal Muscular Atrophy
Spinal Muscular Atrophy and Cardiac Autonomic Function
Respiratory dysfunction is the major cause of morbidity and mortality in patients with spinal muscular atrophy (SMA). Air stacking is a clearance airway technique frequently used but its effects on cardiac autonomic function in…
- Trial name
- NCT01522079
- Testing
- Air stacking manuever
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Centro Universitário Augusto Motta
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Biomarker Development in LGMD2i
The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD2i to support therapeutic development.
- Trial name
- MLB-01-001 (NCT04202627)
- Where
- 11 sites in United States, Denmark
- Volunteers
- 101 people
- Sponsor
- ML Bio Solutions, Inc.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00847379
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 173 people
- Sponsor
- PTC Therapeutics
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