Clinical trials: Atrophy
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
REM Behavior Disorder, Parkinsonism
Efficacy and Tolerability of Ramelteon in Patients With Rapid Eye Movement (REM) Behavior Disorder and Parkinsonism
Parkinson's disease (PD) is the second most common neurodegenerative disorder of the elderly that affects a million patients in US. Sleep dysfunction impacts up to 90% of PD patients.
- Trial name
- NCT00745030
- Testing
- Rozerem
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Northwestern University
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
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
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
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Study of ALS Reversals 4: LifeTime Exposures
Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e.
- Conditions
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
- Trial name
- StARLiTE (NCT03706391)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Duke University
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Biomarker Development for Muscular Dystrophies
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Conditions
- Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
- Trial name
- NCT05019625
- Where
- 5 sites in United States
- Volunteers
- 465 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Vulvovaginal Atrophy, Vasomotor Symptoms
A Phase 1, Open-Label, Parallel Group Study to Evaluate the Pharmacokinetics and Safety of DARE-HRT1 in Healthy PostMenopausal Women
An open-label study to assess the PK of estradiol, estrone and progesterone from the DARE-HRT1 intravaginal rings at two different dose strengths.
- Trial name
- NCT05418426
- Testing
- IVR Dose 1, IVR Dose 2, Oral Reference
- Where
- 2 sites in Australia
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Daré Bioscience, Inc.
Periodontal Disease, Periodontal Bone Loss
Laser Assisted Open Flap Debridement
The study involves the surgical treatment of patients with Stage I to III of periodontitis. Following completion of Steps 1 and 2 of periodontal therapy according to the EFP guidelines for the treatment of periodontitis, sites…
- Trial name
- LaserOpen (NCT07807215)
- Testing
- Mechanical Open Flap debridement, Laser assisted open flap debridement
- Where
- Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Bari Aldo Moro
Duchenne Muscular Dystrophy
Open-label Extension of the HOPE-2 Trial
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up.
- Trial name
- HOPE-2-OLE (NCT04428476)
- Testing
- Deramiocel (CAP-1002)
- Where
- 5 sites in United States
- Volunteers
- 13 people
- Sponsor
- Capricor Inc.
Duchenne Muscular Dystrophy (DMD)
Nutritional Status and Therapy in DMD Patients
Brief Summary The goal of this clinical trial is to evaluate the impact of a disease-specific, individualized diet on the nutritional status and functional abilities of Duchenne muscular dystrophy (DMD) patients.
- Trial name
- NCT06682585
- Testing
- Individualised dietary intervention
- Where
- Turkey (Türkiye)
- Volunteers
- 9 people
- Sponsor
- Ankara University
Spinal Muscular Atrophy (SMA)
Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration.
- Trial name
- STIM-SMA (NCT06977269)
- Testing
- High-frequency repetitive transcranial magnetic stimulation, lumbar puncture
- Where
- Ukraine
- Volunteers
- 20 people
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
Leber's Congenital Amaurosis
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
- Trial name
- NCT03140969
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 11 people
- Sponsor
- Laboratoires Thea
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