Clinical trials: Multi-System-Atrophy
Multiple System Atrophy, Parkinsonism
Potential Use of Autologous and Allogeneic Mesenchymal Stem Cells in Patients With Multiple System Atrophy
The prevalence of Multiple System Atrophy (MSA) is reported to be between 3.4 - 4.9 cases per 100,000 population. The estimated average incidence is 0.6 - 0.7 cases per 100,000 people per year.
- Conditions
- Multiple System Atrophy, Parkinsonism, Multiple System Atrophy, Parkinson Variant
- Trial name
- NCT04876326
- Testing
- Autologous Adipose Mesenchymal Stem Cell Implantation, Allogeneic Umbilical Cord Mesenchymal Stem Cell Implantation, Allogeneic Umbilical Cord Mesenchymal Stem Cell and Adipose Secretome Implantation
- Where
- Indonesia
- Volunteers
- 15 people
- Sponsor
- Universitas Indonesia
Muscular Dystrophy, Duchenne
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
- Trial name
- BRAVE (NCT03400852)
- Testing
- MNK-1411
- Where
- 16 sites in United States, Bulgaria and 6 more
- Volunteers
- 44 people
- Sponsor
- Mallinckrodt ARD LLC
Secondary Progressive Multiple Sclerosis
A Randomised Controlled Trial of Neuroprotection With Lamotrigine in Secondary Progressive Multiple Sclerosis
A present there is no safe treatment for reducing rate at which disability worsens in people with secondary progressive multiple sclerosis.
- Trial name
- NCT00257855
- Testing
- Lamotrigine
- Where
- United Kingdom
- Volunteers
- 120 people
- Sponsor
- University College London Hospitals
Duchenne Muscular Dystrophy (DMD)
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully…
- Trial name
- NCT01540409
- Testing
- AVI-4658 (Eteplirsen)
- Where
- 11 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Multiple System Atrophy
Study of Rifampicin in Multiple System Atrophy
The purpose of this study was to determine whether Rifampicin was effective in slowing or reversing the progression of multiple system atrophy (MSA).
- Trial name
- MSA (NCT01287221)
- Testing
- Rifampicin
- Where
- 10 sites in United States
- Volunteers
- 100 people
- Sponsor
- Phillip Low
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Muscular Atrophy, Spinal
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
- Trial name
- NCT02240355
- Testing
- RO6885247
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 9 people
- Sponsor
- Hoffmann-La Roche
Multiple System Atrophy
Safety and Tolerability of CS10BR05 Inj. in Subjects with Multiple System Atrophy
The purpose of conducting phase 1 trial is to evaluate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells(CS10BR05) in subjects with Multiple System Atrophy.
- Trial name
- NCT03265444
- Testing
- CS10BR05
- Where
- South Korea
- Volunteers
- 9 people
- Sponsor
- Corestemchemon, Inc.
PSP, CBD
Clinico-Pathologic-Genetic-Imaging Study of Neurodegenerative and Related Disorders
The investigators aim to learn more about symptoms suggestive of a neurodegenerative process.
- Conditions
- PSP, CBD, PCA, LPA
- Trial name
- AND1 (NCT04680130)
- Testing
- C-11 PiB, AV1451 Tau, C-11 ER176
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Mayo Clinic
Spinal Muscular Atrophy
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal…
- Trial name
- NCT02644668
- Testing
- Reldesemtiv 150 mg, Reldesemtiv 450 mg
- Where
- 19 sites in United States, Canada
- Volunteers
- 70 people
- Sponsor
- Cytokinetics
DM1, Myotonic Dystrophy 1
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to…
- Conditions
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- MARINA (NCT05027269)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 39 people
- Sponsor
- Avidity Biosciences, Inc.
Autologous Hematopoietic Stem Cell Transplant
Muscle Stimulation for Physical Function During Stem Cell Transplant
Some blood, bone marrow, and lymphatic (hematologic) cancers such as Hodgkin/Non-Hodgkin lymphomas, chronic lymphocytic leukemia, and multiple myeloma, are over-represented in Veterans due to exposures including Agent Orange and…
- Trial name
- NCT04364256
- Testing
- RS-4i Plus Sequential Stimulator (RS Medical, Vancouver, WA)
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- VA Office of Research and Development
Multiple System Atrophy, Cerebellar Variant (Disorder)
tDCS for Multiple System Atrophy With Cerebellar Feature
The aim of study is to investigate most effective site for control the motor coordination using transcranial direct current stimulation in multiple system atrophy with cerebellar feature
- Trial name
- NCT04092556
- Testing
- transcranial direct current stimulation
- Where
- South Korea
- Volunteers
- 10 people
- Sponsor
- Samsung Medical Center
Multi-system Atrophy - Parkinsonian Type, Orthostatic Hypotension, Dysautonomic
Feasibility Study Assessing the Effect of Carbidopa/Levodopa Ratio on Orthostatic Hypotension in Multiple System Atrophy - Parkinsonian Type and Parkinson Disease.
This study is aimed at patients with multi-system atrophy - parkinsonian type (P-MSA) or Parkinson's disease (PD) receiving dopaminergic drugs and suffering from orthostatic hypotension (OH).
- Conditions
- Multi-system Atrophy - Parkinsonian Type, Orthostatic Hypotension, Dysautonomic, Parkinson Disease
- Trial name
- CARBIDOH (NCT06831500)
- Testing
- Administration of Carbidopa/levodopa
- Where
- 2 sites in Switzerland
- Volunteers
- 36 people
- Sponsor
- Julien Bally
Spinal Muscular Atrophy
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
- Trial name
- NCT05824169
- Testing
- GC101
- Where
- 4 sites in China
- Volunteers
- 18 people
- Sponsor
- GeneCradle Inc
Multiple Sclerosis, Tomography, Optical Coherence
Retinal Atrophy and Neurofilament Light Chain in People With Multiple Sclerosis Taking Ofatumumab
This study aims to assess whether, and the degree to which, ofatumumab modulates or reduces rates of retinal atrophy in people with relapsing-remitting MS (RMS), according to baseline serum neurofilament light chain (sNfL) levels.
- Conditions
- Multiple Sclerosis, Tomography, Optical Coherence, Retinal Degeneration, Visual Acuity
- Trial name
- NCT06167642
- Testing
- Ofatumumab
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Johns Hopkins University
Spinal Muscular Atrophy
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age.
- Trial name
- NCT00227266
- Testing
- Valproic Acid and Levocarnitine
- Where
- 6 sites in United States, Canada
- Volunteers
- 94 people
- Sponsor
- University of Utah
Polycystic Kidney Diseases, Polycystic Liver Disease
The Association Between Skeletal Muscle Mass and Severity of Polycystic Liver Disease and Polycystic Kidney Disease
Primary sarcopenia is used to describe aging and progressed with the physiologic decline. Secondary sarcopenia is associated many chronic disease, including acquired immune deficiency syndrome, cancer, chronic heart failure…
- Conditions
- Polycystic Kidney Diseases, Polycystic Liver Disease, Muscle Loss
- Trial name
- NCT05215964
- Testing
- Renal artery embolization
- Where
- Taiwan
- Volunteers
- 600 people
- Sponsor
- National Taiwan University Hospital
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Prostate Cancer
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer.
- Trial name
- ANDROMEDA (NCT07336446)
- Testing
- AZD9750, AZD5305
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 6 people
- Sponsor
- AstraZeneca