Clinical trials: Muscle Atrophy
Duchenne Muscular Dystrophy
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- Trial name
- NCT03373968
- Testing
- Givinostat
- Where
- 39 sites in United States, Belgium and 9 more
- Volunteers
- 206 people
- Sponsor
- Italfarmaco
Sarcopenia
Protein Timing, Lean Mass, Strength and Functional Capacity Gains in Postmenopausal Women
This study evaluated the effect of protein intake immediately after resistance exercise on lean mass, strength, and functional capacity gains in postmenopausal women.
- Trial name
- NCT03372876
- Testing
- Protein and carbohydrate supplementation
- Where
- Brazil
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Federal University of Uberlandia
Facioscapulohumeral Muscular Dystrophy
The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Registry
Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000.
- Trial name
- NCT04001582
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,018 people
- Sponsor
- Newcastle University
Spinal Muscular Atrophy
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in…
- Trial name
- SHINE (NCT02594124)
- Testing
- nusinersen
- Where
- 47 sites in United States, Australia and 12 more
- Volunteers
- 292 people
- Sponsor
- Biogen
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.
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Facioscapulohumeral Muscular Dystrophy
Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
The purpose of this research study is to identify and study changes in muscle in people with facioscapulohumeral muscular dystrophy using magnetic resonance imaging and spectroscopy.
- Trial name
- NCT01671865
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Duchenne Muscular Dystrophy (DMD)
Usability and User Experience of a Virtual Reality Rehabilitation Game Platform in Individuals With Duchenne Muscular Dystrophy
This study aims to evaluate the usability, user experience, and tolerability of a virtual reality-based rehabilitation game platform specifically developed for individuals with Duchenne muscular dystrophy.
- Trial name
- DMD-VR-UE (NCT07565272)
- Testing
- Virtual Reality Gameplay Session
- Where
- Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Istanbul University
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
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
Post-Polio Syndrome
Post-Poliosyndrome Treated With Intravenous Immunoglobulin (IvIg)
Inflammatory cytokines in the cerebrospinal fluid may contribute to the new muscle weakness, fatigue and pain experienced by patients with post-polio syndrome. Intravenousimmunoglobulin (IvIg) reduces this inflammation.
- Trial name
- NCT00231439
- Testing
- IvIg
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Haukeland University Hospital
Spinal Muscular Atrophy
Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen
The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams.
- Trial name
- RetroNusiMFM (NCT04644393)
- Where
- 8 sites in France
- Volunteers
- 40 people
- Sponsor
- Hospices Civils de Lyon
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Trial name
- NCT06280209
- Testing
- BMN 351
- Where
- 7 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Chronic Kidney Disease, Sarcopenia
Effect of Ketosteril on Sarcopenia in Patients With Chronic Kidney Disease
The investigators hypothesize that Ketosteril can improve sarcopenia in patients with renal disease without increasing the burden on the kidneys and causing deterioration of renal function.
- Trial name
- NCT06329622
- Testing
- low-protein diet + Ketosteril, low-protein diet
- Where
- China
- Volunteers
- 58 people
- Sponsor
- Huashan Hospital
Spinal Muscular Atrophy
Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
This prospective interventional study evaluates the immediate and sustained effects of intensive equine-assisted physiotherapy in children with spinal muscular atrophy (SMA).
- Trial name
- HIPOSMA (NCT05341453)
- Testing
- Individual physiotherapy (SMA-SOC-N), Equine-Assisted Physiotherapy Based on Neuroproprioceptive Facilitation and Inhibition, Therapeutic grooming
- Where
- Czechia
- Volunteers
- 21 people
- Sponsor
- Charles University, Czech Republic
Duchenne Muscular Dystrophy
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
This study is single arm, single center trial to study the safety and efficacy of bone marrow-derived autologous specific populations of stem cells and mesenchymal stem cells for the treatment of Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03067831
- Testing
- Stem Cells
- Where
- Jordan
- Volunteers
- 20 people
- Sponsor
- Stem Cells Arabia
Tremor, Involuntary Movements
Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen
Hyperkinetic movement disorders in patients with diseases of motor neurons will be studied. Patients with spinal muscular atrophy (SMA) and motor neuron disease patients will be studied.
- Conditions
- Tremor, Involuntary Movements, SMA, MND (Motor Neurone DIsease)
- Trial name
- NCT04825119
- Testing
- Nusinersen
- Where
- Slovenia
- Volunteers
- 110 people
- Sponsor
- University Medical Centre Ljubljana
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