Clinical trials: Multi-System-Atrophy
Duchenne Muscular Dystrophy (DMD)
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT2-EDO51 (NCT06833931)
- Testing
- IV infusion
- Sponsor
- PepGen Inc
Parkinson Disease, Parkinson's Disease and Parkinsonism
Complex Eye Movements in Parkinson's Disease and Related Movement Disorders
Diagnosing Parkinson's disease (PD) depends on the clinical history of the patient and the patient's response to specific treatments such as levodopa.
- Conditions
- Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, Corticobasal Degeneration
- Trial name
- NCT04925622
- Testing
- Complex eye movement exam
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Saccadous, Inc.
Spinal Cord Injuries, Multiple System Atrophy, Parkinson Variant
Transcutaneous Spinal Cord Stimulation Home Study
The goal of this pilot clinical trial is to examine the safety and feasibility of SCONE as home based therapy for orthostatic hypotension and bowel dysfunction in individuals with spinal cord injury or multiple system atrophy.
- Trial name
- NCT06838637
- Testing
- Spinal COrd NEuromodulation ("SCONE") Device
- Where
- Canada
- Volunteers
- 6 people
- Sponsor
- Michael Berger
Multiple System Atrophy
Clinical Trial to Assess Efficacy, Safety, and Tolerability of Rasagiline Mesylate 1 mg in Patients With Multiple System Atrophy of the Parkinsonian Subtype (MSA-P)
To test the clinical effect of rasagiline on subjects with MSA of the parkinsonian subtype.
- Trial name
- NCT00977665
- Testing
- rasagiline mesylate
- Where
- 47 sites in United States, Austria and 10 more
- Volunteers
- 174 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Transcranial Magnetic Stimulation, Multiple System Atrophy
TMS Treatment in Multiple System Atrophy With Fatigue
Transcranial magnetic stimulation (TMS) is a procedure that has been shown to improve fatigue in chronic sufferers. It uses a plastic covered coil that sends a magnetic pulse through the skull into the brain and by targeting…
- Conditions
- Transcranial Magnetic Stimulation, Multiple System Atrophy, Fatigue
- Trial name
- TMSMSAF (NCT04313530)
- Testing
- Repetitive Transcranial Magnetic Stimulation
- Where
- China
- Volunteers
- 22 people
- Sponsor
- Xuanwu Hospital, Beijing
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Multiple System Atrophy
Fluoxetine in Multiple System Atrophy Patients
This is a French national trial, conducted using a double-blind, placebo-controlled, randomised design involving 15 centers and 88 patients of both sexes.
- Trial name
- MSA-Fluox (NCT01146548)
- Testing
- FLUOXETINE
- Where
- 14 sites in France
- Volunteers
- 87 people
- Sponsor
- University Hospital, Toulouse
Duchenne Muscular Dystrophy
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT01037309
- Testing
- PRO044 SC, PRO044 IV
- Where
- 4 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Parkinsonian Syndrome, Parkinson Disease(PD)
A Study to Evaluate the Diagnostic Efficacy of DaTSCAN™ Ioflupane (123I) Injection in Single Photon Emission Computed Tomography (SPECT) for the Diagnosis of Parkinsonian Syndrome (PS) in Chinese Patients
This is a multicenter, open-label, non-controlled, non-randomized, phase 3 clinical study to compare the SPECT findings after a single IV administration of DaTSCAN™ ioflupane (123I) injection for patients with a clinical…
- Conditions
- Parkinsonian Syndrome, Parkinson Disease(PD), Multiple System Atrophy (MSA), Progressive Supranuclear Palsy (PSP)
- Trial name
- NCT04193527
- Testing
- DaTSCAN™ Ioflupane (123I) Injection
- Where
- 10 sites in China
- Volunteers
- 172 people, healthy volunteers welcome
- Sponsor
- GE Healthcare
Duchenne Muscular Dystrophy
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for…
- Trial name
- NCT01099761
- Testing
- ACE-031 0.5 mg/kg q4wk, ACE-031 1.0 mg/kg q2wk
- Where
- 5 sites in Canada
- Volunteers
- 24 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Multiple System Atrophy
Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins
Multiple System Atrophy (MSA) is a progressive sporadic neurodegenerative disorder leading to widespread loss of brain cells that results in parkinsonian, cerebellar and autonomic dysfunction.
- Trial name
- NCT00750867
- Testing
- intravenous immunoglobulin (IVIg)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Massachusetts, Worcester
Obesity, Muscle Loss
A Study of the Effect of (S)-Pindolol Benzoate (ACM-001.1) on Lean Body Mass (LBM) in Obese Patents During (PROACT 1), and Post-semaglutide Therapy (PROACT 2)
Semaglutide is approved for weight management (weight loss and weight maintenance) in adult obese patients. Drug treatments like semaglutide that result in weight-loss can also decrease muscle mass.
- Trial name
- PROACT (NCT07101939)
- Testing
- (S)-pindolol benzoate, Semaglutide
- Where
- 7 sites in Serbia
- Volunteers
- 120 people
- Sponsor
- Actimed Therapeutics Ltd
Multiple System Atrophy
Cognitive and Behavioral Dysexecutive Syndrome in Multiple System Atrophy
The main objective of the study is to compare the score to the Behavioral Dysexecutive Syndrome Inventory (BDSI) between Parkinsonian Multiple system Atrophy MSA-P patients and cerebellar Multiple System Atrophy (MSA-C) patients…
- Trial name
- CogAMS (NCT02185677)
- Testing
- Clinical evaluation, neuropsychological battery test, MRI
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Bordeaux
Parkinson Disease, MSA - Multiple System Atrophy
Exploring the Olfactory Mucosa, Blood and Urine for the Identification of Early Biomarkers of Parkinson's Disease, Atypical Parkinsonisms and Neurocognitive Disorders Due to Lewy Body Disease
Clinical diagnosis of Parkinson's disease (PD), multiple system atrophy (MSA) and dementia with Lewy bodies (DLB) is challenging, especially in the early stages.
- Conditions
- Parkinson Disease, MSA - Multiple System Atrophy, Lewy Body Dementia (LBD), Neurodegenerative Disease
- Trial name
- EXTRAORDINARY (NCT06846658)
- Where
- 3 sites in Italy
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Healthy Volunteer, Rheumatoid Arthritis
Phenomics in Autoimmune and Inflammatory Diseases
The family of inflammatory/autoimmune systemic diseases (IAD) form a continuum from pure inflammatory diseases to pure autoimmune diseases, encompassing a large panel of inflammatory diseases with some autoimmune components, and…
- Conditions
- Healthy Volunteer, Rheumatoid Arthritis, Ankylosing Spondylitis, Systemic Lupus Erythematosus/Antiphospholipid Syndrome
- Trial name
- TRANSIMMUNOM (NCT02466217)
- Testing
- 1: AID groups, 2: Control groups
- Where
- 2 sites in France
- Volunteers
- 537 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hypotension Symptomatic, Parkinson's Disease
Epidural Electrical Stimulation to Support Hemodynamic Management in Individuals With Parkinson's Disease
The PD-HemON study aims to evaluate the safety and preliminary efficacy of ARC-IM Therapy (Epidural Electrical Stimulation) to support hemodynamic management in people with typical and atypical Parkinson's Disease, who suffer…
- Conditions
- Hypotension Symptomatic, Parkinson's Disease, Orthostatic Hypotension, Dysautonomic, Multiple System Atrophy (MSA) With Orthostatic Hypotension
- Trial name
- PD-HemON (NCT06920134)
- Testing
- ARC-IM Investigational System
- Where
- Switzerland
- Volunteers
- 5 people
- Sponsor
- Ecole Polytechnique Fédérale de Lausanne
Duchenne Muscular Dystrophy (DMD)
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02420379
- Testing
- eteplirsen
- Where
- 13 sites in United States
- Volunteers
- 33 people
- Sponsor
- Sarepta Therapeutics, Inc.
Sarcopenia, Aging
STRIDE: Strength of The Calf Muscles and Their Role In Declining Effectiveness of Movement With Aging
The calf muscles (plantarflexors) are the most important muscles for walking, running and daily activities. Weakness of these muscles through aging and disease significantly impact a person's ability to walk and complete daily…
- Conditions
- Sarcopenia, Aging, Muscle Weakness or Atrophy, Frailty
- Trial name
- STRIDE (NCT07726498)
- Testing
- No intervention (observational study)
- Where
- United Kingdom
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University of Leicester
Wolfram Syndrome
I-Tracking Neurodegeneration in Early Wolfram Syndrome
Wolfram syndrome (WFS; OMIM #222300) is a rare autosomal recessive disease clinically defined in 1938 as the combination of childhood-onset insulin dependent diabetes, optic nerve atrophy, diabetes insipidus and deafness.
- Trial name
- I-TRACK (NCT03951298)
- Where
- United States
- Volunteers
- 128 people
- Sponsor
- Washington University School of Medicine
Multiple System Atrophy
Study of ATH434 in Participants with Multiple System Atrophy
This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy
- Trial name
- NCT05109091
- Testing
- ATH434 dose level 1, ATH434 dose level 2
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 77 people
- Sponsor
- Alterity Therapeutics