Clinical trials: Multi-System-Atrophy
Myotonic Dystrophy
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES…
- Trial name
- NCT06523400
- Testing
- Mexiletine granules for prolonged-release oral suspension
- Where
- 7 sites in Belgium, Denmark and 4 more
- Volunteers
- 176 people
- Sponsor
- Lupin Ltd.
Military Operational Stress Reaction, Malnutrition (Calorie)
Physiological Response to Protein and Energy-enhanced Food Products During Winter Military Training
Soldiers commonly lose muscle mass during training and combat operations that produce large energy deficits (i.e., calories burned > calories consumed).
- Conditions
- Military Operational Stress Reaction, Malnutrition (Calorie), Weight Loss, Muscle Wasting
- Trial name
- NCT05210205
- Testing
- EAA, Energy Dense, Control
- Where
- Norway
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- United States Army Research Institute of Environmental Medicine
Muscular Dystrophy, Duchenne
Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
Placebo-controlled, multi-centre, randomized, double-blind dose escalation study. The aim is to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a…
- Trial name
- NCT02383511
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Multiple System Atrophy
Post-Trial Access to ATH434 for Patients With Multiple System Atrophy
This multicenter post-trial access program is designed to provide ATH434 orally BID to eligible patients who completed Phase 2 studies ATH434-201 or ATH434-202, and who may benefit from this treatment according to the evaluation…
- Trial name
- NCT07531433
- Testing
- ATH434
- Sponsor
- Alterity Therapeutics
Ataxia, Spinocerebellar Ataxias
Transcranial Alternating Current Stimulation (tACS) in Patients With Ataxia
The aim of the study is to evaluate the effects on motor and cognitive performance of transcranial alternating current stimulation (tACS) compared to transcranial direct current stimulation (tDCS) and placebo stimulation (sham)…
- Conditions
- Ataxia, Spinocerebellar Ataxias, Multiple System Atrophy, Ataxia With Oculomotor Apraxia
- Trial name
- SCAtACS (NCT05621200)
- Testing
- Transcranial stimulation over the cerebellar hemispheres
- Where
- Italy
- Volunteers
- 26 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Leber's Hereditary Optic Neuropathy (LHON)
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
The objective of this clinical study is to select the optimal dose and evaluate the safety and efficacy of NR082 in treatment of LHON caused by mitochondrial ND4 gene mutation.
- Trial name
- GOLD (NCT04912843)
- Testing
- NR082 injection
- Where
- China
- Volunteers
- 102 people
- Sponsor
- Wuhan Neurophth Biotechnology Limited Company
Autonomic Nervous System Diseases
Evaluation of Primary Chronic Autonomic Failure
This study will conduct tests in patients with primary chronic autonomic failure (CAF) to learn more about these disorders, which include pure autonomic failure, multiple system atrophy, Parkinson's disease with autonomic…
- Trial name
- NCT00059033
- Where
- United States
- Volunteers
- 325 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Neurodegenerative Diseases, Alzheimer Disease
Sociodemographic Factors and Criminal Behaviour Preceding Neurodegenerative Disease - Retrospective Register Study
In this retrospective register study, clinically classified individuals with neurodegenerative disease from the years 2010-2021 will be verified from the clinical records from KUH and Oulu University Hospital (OUH).
- Conditions
- Neurodegenerative Diseases, Alzheimer Disease, Frontotemporal Dementia, Lewy Body Disease
- Trial name
- DEGERWD (NCT06209515)
- Where
- Finland
- Volunteers
- 61,292 people
- Sponsor
- Kuopio University Hospital
Muscular Dystrophy, Duchenne
Rimeporide in Patients With Duchenne Muscular Dystrophy
In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime.
- Trial name
- RIM4DMD (NCT02710591)
- Testing
- Rimeporide
- Where
- 4 sites in France, Italy and 2 more
- Volunteers
- 20 people
- Sponsor
- EspeRare Foundation
Multiple System Atrophy
Observational Study in Multiple System Atrophy
Talisman is global clinical study (20058N) in Multiple System Atrophy (MSA) patients. It will be conducted in two regions (China and the European Union [EU]).
- Trial name
- TALISMAN (NCT05453058)
- Testing
- plasma NfL and brain MRI (vMRI, DTI, and ASL [if feasible]) only for the EU cohort
- Where
- 8 sites in China
- Volunteers
- 90 people
- Sponsor
- H. Lundbeck A/S
Healthy Volunteers
A First-in-Human Study of Single and Multiple Doses of anle138b in Healthy Subjects
The purpose of this study is to determine the safety, tolerability and blood levels of orally administered anle138b in healthy subjects.
- Trial name
- NCT04208152
- Testing
- anle138b
- Where
- United Kingdom
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- MODAG GmbH
Duchenne Muscular Dystrophy, Cardiomyopathy
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age.
- Trial name
- HOPE (NCT02485938)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Capricor Inc.
Wolfram Syndrome, Diabetes Mellitus
Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study
In this study, the investigators hypothesize that studying monogenic variants with strong effect associated with severe insulin deficiency of Wolfram syndrome will provide important insights into the more complex type 1 and type…
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Deafness
- Trial name
- NCT02841553
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- Washington University School of Medicine
Sarcopenia
Dose Range Finding Study of Bimagrumab in Sarcopenia
The purpose of this study was to determine the efficacy of repeat dosing with multiple dose levels of bimagrumab on patient physical function, skeletal muscle mass and strength in older adults with sarcopenia.
- Trial name
- NCT02333331
- Testing
- bimagrumab
- Where
- 58 sites in United States, Australia and 11 more
- Volunteers
- 217 people
- Sponsor
- Novartis Pharmaceuticals
Neuro-Ophthalmology, Optic Neuropathies
Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study
OCT-A and eco-colordoppler are technologies which enable optic nerve head (ONH) blood perfusion detection. The aim of the study is to assess ONH vascular and structural components in optic neuropathies with and without ONH edema…
- Conditions
- Neuro-Ophthalmology, Optic Neuropathies, Optic Atrophy, Hereditary, Leber, Optic Neuropathy, Anterior Ischemic
- Trial name
- FlowONH (NCT07075887)
- Volunteers
- 70 people
- Sponsor
- Barboni
Muscular Atrophy, Spinal
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
The study is a randomized, single oral dose, crossover study in up to three parts to investigate the relative bioavailability and bioequivalence of two different formulations of risdiplam 5 mg (dispersible tablets) versus the…
- Trial name
- NCT04718181
- Testing
- risdiplam, omeprazole
- Where
- 4 sites in United States
- Volunteers
- 131 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Rheumatoid Arthritis, Ankylosing Spondylitis
PatientSpot Formerly Known as ArthritisPower
Patient Power is a patient research network and database (registry) to collect prospective information about demographics, self-reported diagnoses and medications, and willingness to participate in research from participants with…
- Conditions
- Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout
- Trial name
- NCT03840928
- Where
- United States
- Volunteers
- 40,000 people, healthy volunteers welcome
- Sponsor
- Global Healthy Living Foundation
Parkinson Disease, Multiple System Atrophy
Pain and Autonomic Symptoms in Parkinson's Disease and Atypical Parkinsonisms
The goal of this observational study is to learn about the impact of the different types of pain and of the domains involved in the autonomic disorders of inpatients and outpatients diagnosed with Parkinson disease (PD) and…
- Trial name
- NCT05748028
- Testing
- Rehabilitation, Administration of Clinical scales
- Where
- 9 sites in Italy
- Volunteers
- 280 people
- Sponsor
- Istituti Clinici Scientifici Maugeri SpA
Myotonic Dystrophy 1
Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)
Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystem disorder caused by an expanded CTG trinucleotide repeat in the 3' untranslated region of the DMPK gene.
- Trial name
- C-DMCOS-DM1 (NCT06101940)
- Testing
- MRI scan, Electrocardiography, Pulmonary function test
- Where
- 22 sites in China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Huashan Hospital
Progressive Supranuclear Palsy, Dementia With Lewy Bodies
In-Home Care for Patients With PSP and Related Disorders
Progressive Supranuclear Palsy and related disorders (PRD) are debilitating, costly, and understudied conditions. Improving access to comprehensive, specialized, in-home patient care offers the potential to minimize the downward…
- Conditions
- Progressive Supranuclear Palsy, Dementia With Lewy Bodies, Multiple System Atrophy, Corticobasal Syndrome
- Trial name
- NCT03552484
- Testing
- Home Visit Program, Usual Care/Online Survey
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Rush University Medical Center