Clinical trials: Multi-System-Atrophy
Duchenne Muscular Dystrophy
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02056808
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Sarcopenia
Study of the Safety and Efficacy of REGN1033 (SAR391786) in Patients With Sarcopenia
This is a randomized, double-blind, placebo-controlled, multicenter phase 2 study of the safety and efficacy of 3-month subcutaneous (SC) REGN1033 (SAR391786) treatment in patients with sarcopenia.
- Trial name
- NCT01963598
- Testing
- REGN1033 (SAR391786)
- Where
- 43 sites in United States, France and 2 more
- Volunteers
- 253 people
- Sponsor
- Regeneron Pharmaceuticals
Parkinson Disease, Multiple System Atrophy
The Effect of Gluten-free Diet on Parkinsonism
Recent data suggest that the brain-gut axis, chronic intestinal inflammation and microbiome may contribute to the pathogenesis of neurodegenerative diseases with alfa-synucleinopathy, which include Parkinson's disease (PD) and…
- Conditions
- Parkinson Disease, Multiple System Atrophy, Non-celiac Gluten Sensitivity
- Trial name
- GFREEPARK (NCT05238545)
- Testing
- gluten-free diet
- Where
- Czechia
- Volunteers
- 90 people
- Sponsor
- General University Hospital, Prague
Multiple System Atrophy
Comprehensive Swallowing Rehabilitation in Patients With MSA
The purpose of this study is to investigate the effect of comprehensive swallowing rehabilitation in patients with multiple system atrophy.
- Trial name
- NCT04782284
- Testing
- Comprehensive swallowing rehabilitation, Swallowing education
- Where
- South Korea
- Volunteers
- 24 people
- Sponsor
- Seoul National University Hospital
MSA - Multiple System Atrophy
MotIoN aDaptive Deep Brain Stimulation for MSA
Patients routinely undergo deep brain stimulation (DBS) for treatment of symptoms related to neurodegenerative conditions, most commonly Parkinson's disease.
- Trial name
- MINDS (NCT05197816)
- Testing
- Deep brain stimulation
- Where
- United Kingdom
- Volunteers
- 3 people
- Sponsor
- University of Oxford
Multiple System Atrophy
Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophy
The present study aims to conduct a randomized controlled trial to evaluate the efficacy and safety of 3-n-Butylphthalide (NBP) in improving symptoms in patients with Multiple System Atrophy (MSA).
- Trial name
- ENMSA (NCT07518810)
- Testing
- 3-N-butylphthalide
- Where
- 7 sites in China
- Volunteers
- 150 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Parkinson Disease, Progressive Supranuclear Palsy
Speech Accessibility Project
The goal of the Speech Accessibility Project at the University of Illinois Beckman Institute (https://speechaccessibilityproject.beckman.illinois.edu) is to collect, annotate, and curate a shared database of speech samples from…
- Conditions
- Parkinson Disease, Progressive Supranuclear Palsy, Multiple System Atrophy, Dementia With Lewy Bodies
- Trial name
- SAP (NCT05889260)
- Where
- 2 sites in United States
- Volunteers
- 485 people
- Sponsor
- University of Illinois at Urbana-Champaign
Prader Labhart Willi Syndrome, Body Weight Changes
Prader-Willi Syndrome Body Composition
Adults aged over 18 years, with the diagnosis of Prader-willi syndrome will be recruited through the hospital's outpatient clinic for either as intervention group receiving therapeutic elastic band training, or as control group…
- Conditions
- Prader Labhart Willi Syndrome, Body Weight Changes, Sarcopenia
- Trial name
- NCT04597645
- Testing
- Therapeutic elastic band resistance training
- Where
- Taiwan
- Volunteers
- 20 people
- Sponsor
- Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation
Duchenne Muscular Dystrophy
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN…
- Trial name
- NCT02958202
- Testing
- BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 7 people
- Sponsor
- BioMarin Pharmaceutical
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II
Spinraza in Adult Spinal Muscular Atrophy
This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type 3
- Trial name
- SAS (NCT03709784)
- Testing
- Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey
- Where
- 11 sites in United States, Canada
- Volunteers
- 148 people
- Sponsor
- Washington University School of Medicine
Parkinson Disease, Multiple System Atrophy, Parkinson Variant
Abdominal Binders to Treat Orthostatic Hypotension in Parkinsonian Syndromes
The purpose of the present clinical trial is to determine whether the use of an elastic abdominal binder is effective in the non-pharmacological management of symptomatic, neurogenic orthostatic hypotension (OH) in individuals…
- Conditions
- Parkinson Disease, Multiple System Atrophy, Parkinson Variant, Orthostatic; Hypotension, Neurogenic
- Trial name
- ABOH-PS (NCT04920552)
- Testing
- Elastic abdominal binder
- Where
- Austria
- Volunteers
- 30 people
- Sponsor
- Universitätsklinik für Neurologie, Innsbruck
Duchenne Muscular Dystrophy
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
- Trial name
- NCT02251600
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Spinal Muscle Atrophy
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with…
- Trial name
- NCT07531719
- Where
- 2 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Osteoporosis, Osteoporotic Fractures
Multifaceted Intervention Using Telehealth to Reduce the Risk of Falls and Fractures in Older Men
Men sustain over one-third of osteoporosis-related fractures worldwide. The burden of osteoporotic fractures in older men is substantial, and men suffer significantly worse fracture-related outcomes than women.
- Conditions
- Osteoporosis, Osteoporotic Fractures, Osteoporosis, Age-Related, Muscle Loss
- Trial name
- MisterFIt (NCT05927623)
- Testing
- Multifaceted Virtual Fracture Prevention Program, Attention Control
- Where
- 5 sites in Canada
- Volunteers
- 45 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
PSP, PSP - Progressive Supranuclear Palsy
The CurePSP Genetics Program
This study is an observational, prospective genetic study. It aims to obtain DNA for research and testing from patients with PSP, CBS, MSA, and related neurological conditions and their families.
- Conditions
- PSP, PSP - Progressive Supranuclear Palsy, Corticobasal Syndrome, Corticobasal Syndrome(CBS)
- Trial name
- NCT06647641
- Testing
- Whole genome sequencing will be performed at the NIH
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Duchenne Muscular Dystrophy
First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT06565208
- Testing
- SAT-3247
- Where
- 2 sites in Australia
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- Satellos Bioscience, Inc.
MSA
Mesenchymal Stem Cell Therapy in Multiple System Atrophy
The purpose of this study is to determine whether mesenchymal stem cells (MSCs) can be safely delivered to the cerebrospinal fluid (CSF) of patients with multiple system atrophy (MSA). Funding Source - FDA OOPD.
- Trial name
- NCT02315027
- Testing
- Autologous Mesenchymal Stem Cells
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Mayo Clinic
Parkinson's Disease, Multiple System Atrophy, Parkinson Variant
AB-1005 Long-Term Follow-up Study
This is an observational, long term, follow-up study for participants with Parkinson's disease (PD) or Multiple System Atrophy-Parkinsonian subtype (MSA-P) who participated in prior interventional studies with AB-1005.
- Trial name
- NCT07081841
- Testing
- Observation
- Where
- 2 sites in United States
- Volunteers
- 132 people
- Sponsor
- AskBio Inc
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Spinal Muscular Atrophy (SMA)
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which…
- Trial name
- MANATEE (NCT05115110)
- Testing
- RO7204239, Risdiplam
- Where
- 30 sites in United States, Australia and 10 more
- Volunteers
- 97 people
- Sponsor
- Hoffmann-La Roche