Clinical trials: Multi-System-Atrophy
Multiple System Atrophy, Progressive Supranuclear Palsy (PSP)
Synaptic Loss in Multiple System Atrophy
In this study the investigators would like to investigate the degree of damage of the synapses, an important part of the neurons vital for the communications between neurons, in Multiple System Atrophy (MSA), and pathology…
- Trial name
- NCT05121012
- Testing
- Study procedures for MSA patients, Study procedures for PSP patients
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- University of Exeter
Wolfram Syndrome, Fecal Incontinence
Mechanisms of Fecal Incontinence in Wolfram Syndrome
In order to clarify the mechanism of fecal incontinence in in Wolfram syndrome and to characterize its risk factors, the examiner will reviewed the files of patients followed in a Reference Center for Rare Diseases in…
- Trial name
- NCT07313085
- Testing
- Retrospective case-control analysis
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hôpital Necker-Enfants Malades
Autonomic Failure, Pure Autonomic Failure
CPAP for the Treatment of Supine Hypertension
This study aims to learn about the effects of continuous positive airway pressure (CPAP) on people with autonomic failure and high blood pressure when lying down (supine hypertension) to determine if it can be used to treat their…
- Conditions
- Autonomic Failure, Pure Autonomic Failure, Multiple System Atrophy, Parkinson Disease
- Trial name
- NCT05489575
- Testing
- Active CPAP, Sleeping in a head-up tilt (HUT) position
- Where
- United States
- Volunteers
- 59 people
- Sponsor
- Vanderbilt University Medical Center
Sarcopenia in Elderly
A Study of PLH-2301 in Subjects With Sarcopenia
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of PLH-2301 in subjects with sarcopenia.
- Trial name
- PLH-2301_P2 (NCT07390552)
- Testing
- PLH-2301
- Where
- 5 sites in South Korea
- Volunteers
- 168 people
- Sponsor
- Pluto Inc.
Multiple System Atrophy
Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France
This multicenter open-label extension study conducted in France is designed to provide ATH434 orally BID to eligible patients who completed the Phase 2 study ATH434-201, and who may benefit from this treatment according to the…
- Trial name
- NCT07729852
- Testing
- ATH434
- Where
- 4 sites in France
- Volunteers
- 7 people
- Sponsor
- Alterity Therapeutics
Myotonic Dystrophy 1
Study of ATX-01 in Participants With DM1
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated.
- Trial name
- ArthemiR (NCT06300307)
- Testing
- ATX-01
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 56 people
- Sponsor
- ARTHEx Biotech S.L.
Parkinson Disease, Essential Tremor
Brain Network Activation in Patients With Movement Disorders
The diagnosis and management of movement disorders, such as Parkinson's disease (PD), parkinson-plus syndromes (PPS), dystonia, essential tremor (ET), normal pressure hydrocephalus (NPH) and others is challenging given the lack…
- Conditions
- Parkinson Disease, Essential Tremor, Dystonia, Normal Pressure Hydrocephalus
- Trial name
- BNA-MDi (NCT03269201)
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- Sheba Medical Center
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
Parkinson's Disease, Parkinsonism
Modifiable Variables in Parkinsonism (MVP)
We are trying to identify factors associated with improved quality of life and fewer PD symptoms. We are attempting to identify practices, beliefs, and therapies used by individuals who report excellent quality of life, few PD…
- Conditions
- Parkinson's Disease, Parkinsonism, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- NCT02194816
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- Bastyr University
Multiple System Atrophy, Orthostatic Hypotension
Reduction in Splanchnic Capacitance Contributes to Sympathetically Dependent Hypertension in Autonomic
The purpose of this study is to learn more about the regulation of the veins of the abdomen by the autonomic (involuntary) nervous system, and how this may affect high blood pressure.
- Conditions
- Multiple System Atrophy, Orthostatic Hypotension, Supine Hypertension
- Trial name
- NCT02726711
- Testing
- Trimethaphan
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Vanderbilt University
Retina Detachment, Eye Injuries
Multi-center Clinical Observation of FCVB in Guangdong Province
This study provides further theoretical guidance for clinical application of FCVB through observing and collecting various indicators before and after surgical treatment of the FCVB in the real world.
- Conditions
- Retina Detachment, Eye Injuries, Eye Atrophy, Silicone Emulsion
- Trial name
- NCT03908515
- Testing
- Foldable Capsular Vitreous Body (FCVB)
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Sun Yat-sen University
Parkinson's Disease, Alzheimer's Disease
Biomarkers in Neural Disorders
This study seeks to establish the sensitivity and specificity of what appears to be a unique brainstem biomarker of Parkinson's Disease (PD) - an electrically induced olygosynaptic nasotrigeminal reflex response - in…
- Conditions
- Parkinson's Disease, Alzheimer's Disease, Progressive Supranuclear Palsy, Essential Tremor
- Trial name
- NCT02761707
- Testing
- Electrical Brainstem Responses, Olfactory Tests, Visual Deprivation
- Where
- United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Amyotrophic Lateral Sclerosis, Brainstem Stroke
Optimizing BCI-FIT: Brain Computer Interface - Functional Implementation Toolkit
This project adds to non-invasive BCIs for communication for adults with severe speech and physical impairments due to neurodegenerative diseases.
- Conditions
- Amyotrophic Lateral Sclerosis, Brainstem Stroke, Muscular Dystrophies, Parkinson's Disease and Parkinsonism
- Trial name
- BCI-FIT (NCT04468919)
- Testing
- BCI-FIT multi-modal access, BCI-FIT adaptive signal modeling, BCI-FIT active querying
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
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
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-44 (NCT07037862)
- Testing
- ENTR-601-44
- Where
- 14 sites in Belgium, Italy and 2 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Cerebral Palsy, Down Syndrome
Investigation of Parents' Anxiety Level and Health Related Quality of Life in Different Types of Physical Disabilities
Disability brings many psychosocial problems in society. The effects of the health of a disabled child on the psychological health and quality of life of the family are inevitable.
- Conditions
- Cerebral Palsy, Down Syndrome, Spina Bifida, Muscular Dystrophy
- Trial name
- NCT04035967
- Testing
- Questionnaire
- Where
- Turkey (Türkiye)
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Sanko University
Protein Deficiency, Training Group, Sensitivity
Effects of a Recovery Supplement on Body Composition, Performance, Muscular Properties, and Wellbeing
The investigation will be conducted as a double blinded, randomized, parallel between treatment conditions comparison design with two different groups ingesting a different supplement each one.
- Conditions
- Protein Deficiency, Training Group, Sensitivity, Fatigue, Muscular Atrophy
- Trial name
- NCT05769101
- Testing
- Ingestion of a Post-Workout Supplement, Ingestion of a Maltodextrin Supplement
- Where
- United Kingdom
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Joel Puente
Multiple System Atrophy (MSA)
A Phase 2 Study of ONO-2808 in Patients With Multiple System Atrophy
This is a Phase 2, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of multiple doses of ONO-2808 in patients with MSA.
- Trial name
- NCT05923866
- Testing
- ONO-2808
- Where
- 35 sites in United States, Japan
- Volunteers
- 92 people
- Sponsor
- Ono Pharmaceutical Co., Ltd.
Spinal Muscular Atrophy Type 3
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
- Trial name
- NCT06421831
- Testing
- GC101
- Where
- China
- Volunteers
- 21 people
- Sponsor
- GeneCradle Inc
Healthy Volunteer, Muscular Atrophy, Spinal
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
- Trial name
- NCT05575011
- Testing
- BIIB115
- Where
- 16 sites in Belgium, Canada and 7 more
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Biogen