Clinical trials: Multi-System-Atrophy
Neurogenic Orthostatic Hypotension, Autonomic Failure
Effect of Midodrine vs Abdominal Compression on Cardiovascular Risk Markers in Autonomic Failure Patients
The purpose of this study is to learn more about the effects of abdominal compression and the medication midodrine, two interventions used for the treatment of orthostatic hypotension (low blood pressure on standing), on…
- Conditions
- Neurogenic Orthostatic Hypotension, Autonomic Failure, Pure Autonomic Failure, Multiple System Atrophy
- Trial name
- NCT04620382
- Testing
- Midodrine, Abdominal compression
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Vanderbilt University Medical Center
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Multiple System Atrophy, Parkinsons Disease
Autologous suraL nervE Grafting to the Substantia nigrA in Patients With Synuclienopathies
This phase I double-blind study focuses on the safety and feasibility of implanting autologous peripheral nerve tissue (PNT) into the substantia nigra area of the brain in persons who have been diagnosed with either Parkinson's…
- Trial name
- LEAP (NCT06683365)
- Testing
- Sural Nerve Graft to the Substantia Nigra
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Craig van Horne, MD, PhD
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
Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
This study aims to characterize the pathophysiological mechanisms of 21 different metabolic myopathies. The study will focus on exercise capacity and the metabolic derangement during exercise.
- Conditions
- Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors, Carbohydrate Metabolism, Inborn Errors, Long-Chain 3-Hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT02635269
- Testing
- Sugar
- Where
- Denmark
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
This prospective observational study aims to identify and preliminarily validate molecular biomarkers, including microRNAs and metabolites, for the early differentiation of Parkinson's disease (PD) from atypical parkinsonian…
- Conditions
- PARKINSON DISEASE (Disorder), Atypical Parkinsonism, Multiple System Atrophy, Progressive Supranuclear Palsy (PSP)
- Trial name
- BIOMARK-PS (NCT07604883)
- Where
- 2 sites in Poland
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- International Institute of Molecular and Cell Biology in Warsaw
Multiple System Atrophy
Medical Decision Making in Multiple System Atrophy
The goal of this clinical study is to evaluate the effects of a personalized symptomatic treatment plan integrated with monthly telemedicine and mobile palliative care interventions on a population of individuals diagnosed with…
- Trial name
- MeDeMSA (NCT06072105)
- Testing
- Telemedicine visits, Multidisciplinary, personalized symptomatic treatment
- Where
- Austria
- Volunteers
- 92 people
- Sponsor
- Medical University Innsbruck
Hypertension, Pure Autonomic Failure
Overnight Trials With Heat Stress in Autonomic Failure Patients With Supine Hypertension
Patients with autonomic failure are characterized by disabling orthostatic hypotension (low blood pressure on standing), and at least half of them also have high blood pressure while lying down (supine hypertension).
- Conditions
- Hypertension, Pure Autonomic Failure, Multiple System Atrophy, Autonomic Failure
- Trial name
- NCT03042988
- Testing
- Heating pad
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Vanderbilt University Medical Center
Supine Hypertension, Autonomic Failure
Treatment of Supine Hypertension in Autonomic Failure (CPAP)
Supine hypertension is a common problem that affects at least 50% of patients with primary autonomic failure. Supine hypertension can be severe and complicates the treatment of orthostatic hypotension.
- Trial name
- NCT03312556
- Testing
- continuous positive airway pressure (CPAP)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Vanderbilt University Medical Center
Frailty, Sarcopenia
Development of Continuous Assessment of Frailty and Muscle Quality in Older Subjects: ECOFRAIL Protocol
We are conducting a study in different centers in Spain in elderly people, in order to assess the effectiveness of a physical exercise intervention program in people in a situation of frailty.
- Conditions
- Frailty, Sarcopenia, Ultrasonography, Exercise
- Trial name
- ECOFRAIL (NCT05294757)
- Testing
- Physical exercise
- Where
- 3 sites in Spain
- Volunteers
- 85 people, healthy volunteers welcome
- Sponsor
- University of Deusto
Neurodegenerative Diseases, Parkinson Disease
Neurodegenerative Diseases Registry
With the increase in life expectancy of our population due to advancement of medical diagnosis and treatments, the incidence of age dependent neurodegenerative diseases increased, including Alzheimer's disease (AD), parkinsonian…
- Conditions
- Neurodegenerative Diseases, Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- NDD Registry (NCT04472130)
- Where
- Hong Kong
- Volunteers
- 1,400 people, healthy volunteers welcome
- Sponsor
- Vincent Mok
Multiple Sclerosis
Effects of a Weight Based Training Program on MS Patients
Low bone density is a health risk in older adults and especially people with multiple sclerosis (MS) due to steroid treatments and less mobility. Bone density is a measurement of how dense or strong bones are.
- Trial name
- NCT04002492
- Testing
- Bodyweight training
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Holy Name Medical Center, Inc.
Myotonic Dystrophy Type 1
A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1
This study will test the safety, tolerability, and pharmacokinetics of multiple escalating doses of ISIS-DMPKRx administered subcutaneously to adult patients with DM1.
- Trial name
- NCT02312011
- Testing
- IONIS-DMPKRx
- Where
- 8 sites in United States
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Facio-Scapulo-Humeral Dystrophy, FSHD2
Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
This multi-centre, randomised, double-blind, placebo-controlled crossover trial will compare changes in strength-related motor function following treatment with creatine monohydrate to treatment with placebo, as measured by the…
- Conditions
- Facio-Scapulo-Humeral Dystrophy, FSHD2, FSHD1
- Trial name
- NCT02948244
- Testing
- Creatine Monohydrate
- Where
- Australia
- Volunteers
- 11 people
- Sponsor
- Murdoch Childrens Research Institute
Post Polio Syndrome, PPS
Efficacy and Safety of Xepol (Human Immunoglobulin) in Subjects With Post-Polio Syndrome (PPS)
The primary objective was to assess the effect of Xepol compared to placebo on physical health and on muscle strength in subjects with post-polio syndrome.The secondary objective was to assess the effect of Xepol compared to…
- Trial name
- NCT00160082
- Testing
- Xepol
- Where
- 4 sites in Sweden
- Volunteers
- 124 people
- Sponsor
- Calliditas Therapeutics AB
Multiple System Atrophy
"An Intermediate Size Patient Population Expanded Access Protocol to Evaluate the Safety and Efficacy of Allogeneic HB-adMSCs (Hope Biosciences - Adipose Derived Mesenchymal Stem Cells) for the Treatment of Multiple System Atrophy."
This expanded access protocol is part of IND 32226 to evaluate efficacy and safety of multiple intravenous administrations of allogeneic HB-adMSCs for the treatment of Multiple System Atrophy for up to 7 adult patients who pass…
- Trial name
- NCT07238062
- Testing
- HB-adMSCs - Hope Biosciences Adipose Derived Mesenchymal Stem Cells
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Parkinson Disease (PD), MSA - Multiple System Atrophy
Test-retest Trial With [11C]MODAG-005 in PD or MSA and AMHC - Pilot Phase
This is an open-label, single-center Phase 1 study evaluating the safety, tolerability, and test-retest characteristics of [11C]MODAG-005, an investigational positron emission tomography/computed tomography (PET/CT) radioligand…
- Conditions
- Parkinson Disease (PD), MSA - Multiple System Atrophy, Healthy Adult Participants
- Trial name
- PIOSA (NCT07640542)
- Testing
- [11C]MODAG-005
- Where
- Germany
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- MODAG GmbH
Ventilator Induced Diaphragm Dysfunction
A Protocol Comparing Temporary Transvenous Diaphragm Pacing to Standard of Care for Weaning From Mechanical Ventilation
This clinical investigation is an open-label, multi-center RCT to demonstrate the safety and effective performance of the Lungpacer DPTS (plus standard of care) as compared to Control (standard of care only) in patients aged 18…
- Trial name
- RESCUE 3 (NCT03783884)
- Testing
- Lungpacer Diaphragm Pacing Therapy
- Where
- 33 sites in United States, France and 1 more
- Volunteers
- 223 people
- Sponsor
- Lungpacer Medical Inc.
Parkinson Disease, Osteoporosis
Trial of Parkinson's And Zoledronic Acid
This home-based study is a randomized (1:1) placebo-controlled trial of a single infusion of zoledronic acid-5 mg (ZA) for the prevention of fractures in men and women aged 60 years and older with Parkinson's disease and…
- Conditions
- Parkinson Disease, Osteoporosis, Parkinsonism, Parkinson's Disease and Parkinsonism
- Trial name
- TOPAZ (NCT03924414)
- Testing
- Zoledronic Acid 5Mg/Bag 100Ml Inj
- Where
- 77 sites in United States
- Volunteers
- 2,721 people
- Sponsor
- California Pacific Medical Center Research Institute
Multiple Sclerosis
Analysis of Neurodegenerative Process Within Visual Ways In Multiple Sclerosis
This study will interest in the pathophysiology of silent retinal axonal loss in multiple sclerosis. Recent studies have suggested that silent retinal axonal loss (no past history of optic neuritis [ON]) may be due to…
- Trial name
- VWIMS (NCT03656055)
- Where
- France
- Volunteers
- 108 people
- Sponsor
- University Hospital, Lille