Treatment Trials

99 Clinical Trials for Various Conditions

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A Phase 2a Study of Foralumab Nasal in Patients with Multiple System Atrophy (MSA)
Description

A Phase 2a Study of Foralumab Nasal in Patients with Multiple System Atrophy (MSA)

Conditions

Multiple System Atrophy

A Phase 2 Study of YA-101 in Patients with Multiple System Atrophy
Description

This is a Phase 2, double-blind, placebo-controlled, multi-center, Phase II, dose escalation study to evaluate the safety, tolerability, pharmacokinetics and efficacy of Ya-101 in subjects with multiple system atrophy.

Conditions

Multiple System Atrophy

A Trial of Lu AF82422 in Participants With Multiple System Atrophy (MSA)
Description

The main goal of this trial is to evaluate the efficacy and safety of Lu AF82422 for the treatment of participants with Multiple System Atrophy (MSA).

Conditions

Multiple System Atrophy

A Study to Test if TEV-56286 is Effective in Relieving Multiple System Atrophy Safety and Efficacy Study
Description

The primary objective of the study is to evaluate the efficacy of TEV-56286 administered orally for the treatment of adult participants with Multiple System Atrophy (MSA). A secondary objective of the study is to evaluate specific efficacy parameters of TEV-56286. Another secondary objective is to evaluate the safety and tolerability of TEV-56286. The planned study period per participant is 56 weeks including a screening period (up to 4 weeks), a 48-week double-blind treatment period, and a follow-up visit (approximately 4 weeks after the end of the double-blind treatment period). The study duration will be approximately 27 months.

Conditions

Multiple System Atrophy

A Phase 2 Study of ONO-2808 in Patients With Multiple System Atrophy
Description

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. This is the first study of ONO-2808 in patients with MSA.

Conditions

Multiple System Atrophy (MSA)

Pre-Gene Therapy Study in Parkinson's Disease and Multiple System Atrophy
Description

The objective of this study is to describe disease progression in study participants diagnosed with early Parkinson's Disease or Multiple System Atrophy - Parkinsonian Type up to 18 months as delineated by clinical and biochemical parameters.

Conditions

Parkinson's Disease, Multiple System Atrophy, Parkinson Variant, Multiple System Atrophy

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic NOH in Participants with Multiple System Atrophy
Description

This is a Phase 3, multi-center, randomized withdrawal study to evaluate the efficacy and durability of ampreloxetine in participants with MSA and symptomatic nOH after 20 weeks of treatment. This study includes 4 periods: Screening, open label, randomized withdrawal, and long-term treatment extension (LTE).

Conditions

Symptomatic Neurogenic Orthostatic Hypotension, MSA - Multiple System Atrophy

A Study of TAK-341 in Treatment of Multiple System Atrophy
Description

The main aim is to see how TAK-341 works after 52 weeks in participants with multiple system atrophy as measured by the Unified Multiple System Atrophy Rating Scale Part I (UMSARS). The study will enroll approximately 138 patients. Participants will receive a total of 13 intravenous infusions every 4 weeks approximately, these may be either of TAK-341 or placebo, after each infusion some blood samplings will be taken and other assessments completed. This trial will be conducted in North America, Europe and Asia.

Conditions

Multiple System Atrophy

Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophy
Description

Multiple system atrophy (MSA) is a rare, rapidly progressive, and invariably fatal neurological condition characterized by autonomic failure, parkinsonism, and/or ataxia. There is no available treatment to slow or halt disease progression. The purpose of this study is to assess optimal dosing frequency, effectiveness and safety of adipose-derived autologous mesenchymal stem cells delivered into the spinal fluid of patients with MSA. Funding source: FDA Office of Orphan Product Development (OOPD), Mayo Clinic Executive Dean for Research Transformational Award, Mayo Clinic Regenerative Medicine, and Mayo Clinic Department of Neurology.

Conditions

Multiple System Atrophy

Study of ATH434 in Participants with Multiple System Atrophy
Description

This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy

Conditions

Multiple System Atrophy

A Study of Lu AF82422 in Participants With Multiple System Atrophy
Description

To find out the effect of Lu AF82422 on disease progression in participants with multiple system atrophy.

Conditions

Multiple System Atrophy

Expanded Access Protocol of Verdiperstat in Patients With Multiple System Atrophy (MSA)
Description

The purpose of this expanded access program is to provide access to the investigational drug verdiperstat in patients with Multiple System Atrophy (MSA). Expanded access allows patients with a serious or a life-threatening disease or condition access to an investigational drug when no satisfactory approved treatment options are available.

Conditions

Multiple System Atrophy (MSA)

GDNF Gene Therapy for Multiple System Atrophy
Description

The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.

Conditions

Multiple System Atrophy

TRACK-MSA: A Longitudinal Study to Define Outcome Measures in Multiple System Atrophy
Description

TRACK-MSA is an observational, non-interventional, longitudinal natural history study to define changes in clinical, neurological, blood, CSF, and neuroimaging biomarkers in patients with multiple system atrophy (MSA) comparing baseline to 6-month and 1-year assessments. The study will enroll 50 patients with MSA-P or MSA-C at 2 or more participating sites.

Conditions

Multiple System Atrophy

Study of BHV-3241 in Participants With Multiple System Atrophy
Description

The purpose of this study is to compare the efficacy of verdiperstat (BHV-3241) versus placebo in participants with Multiple System Atrophy

Conditions

Multiple System Atrophy

Multiple System Atrophy Multidisciplinary Clinic
Description

This is a prospective cohort study to examine the disease burden of multiple system atrophy and the impact of multidisciplinary care on quality of life and caregiver burden. Data will be collected through valid rating scales completed by patients and caregivers at home or in the MSA clinic.

Conditions

Multiple System Atrophy (MSA)

A Futility Trial of Sirolimus in Multiple System Atrophy
Description

Single-center, randomized, placebo-controlled, phase-II, futility clinical trial to determine if oral sirolimus is of sufficient promise to slow disease progression in MSA, prior to embarking on a large-scale and costly phase III study to assess its efficacy. A futility design under the null hypothesis assumes that sirolimus will slow the progression of the disease, whereas the alternative hypothesis assumes no benefit of sirolimus. If the null hypothesis is rejected (i.e., futility of sirolimus to slow progression of MSA), a major phase III study will be discouraged, whereas non-futility will offer strong support for a phase III trial to detect clinical efficacy.

Conditions

Multiple System Atrophy

Mesenchymal Stem Cell Therapy in Multiple System Atrophy
Description

The purpose of this study is to determine whether mesenchymal stem cells (MSCs) can be safely delivered to the cerebrospinal fluid (CSF) of patients with multiple system atrophy (MSA). Funding Source - FDA OOPD.

Conditions

MSA

Defining Phenotypes of Movement Disorders :Parkinson's Plus Disorders (PD), Essential Tremor (ET), Cortical Basal Degeneration (CBD), Multiple Systems Atrophy (MSA), Magnetoencephalography.
Description

Investigators hypothesize that there are specific characteristic of each cognitive and motor condition that can be defined using brains scans.

Conditions

Essential Tremor, Multiple System Atrophy, Corticobasal Degeneration, Supranuclear Palsy, Progressive, Parkinson Disease

Treatment of Parkinson Disease and Multiple System Atrophy Using Intranasal Insulin.
Description

Parkinson disease (PD) and multiple system atrophy (MSA) are progressive neurodegenerative disorders characterized by abnormal accumulation of α-synuclein. There is no effective treatment that can slow down the disease progression and both disorders are associated with severe cognitive decline. It was shown that intranasal insulin (INI) improves learning and memory in healthy and cognitively impaired non-diabetic adults. The proof-of-concept, randomized, placebo-controlled, cross-over pilot study ( NCT01206322) has shown that a single 40 international units dose of intranasal insulin improves visuospatial memory in diabetes and control subjects. This proposal includes randomized, double blinded, placebo-controlled trial of intranasal insulin (40 international units daily) in treatment of PD and MSA. The study will evaluate 22 patients with PD and 22 patients with MSA. Total duration of the study will be 2 years. The primary goal is to assess the efficacy of INI in treatment of cognitive abnormalities in both PD and MSA. The primary efficacy end point will be change of the cognitive scale ratings.

Conditions

Parkinson Disease, Multiple System Atrophy

PET Imaging Study of Neurochemical and Autonomic Disorders in Multiple System Atrophy (MSA)
Description

Multiple system atrophy (MSA) is a disorder of the nervous system of unclear cause. In MSA there is degeneration (progressive loss) of nerve cells in several brain and spinal cord regions. The result is a variety of symptoms, from physical (parkinsonism, ataxia, incoordination, falls, slowness) to autonomic (fainting, bladder incontinence, sexual dysfunction) to sleep problems (dream enactment, sleep apnea). This research aims to help us better understand the patterns and timing of nerve degeneration relatively early in the disease, and how this affects symptoms and progression. For instance: 1. Does MSA affect certain nerves that stimulate heart pumping? If so, does the severity of loss of heart nerves affect disease progression and survival? 2. It is thought that MSA does not affect memory and thinking much, unlike other diseases (such as Parkinson's). Is this accurate? Is there loss of nerves that transmit acetylcholine (a neurochemical important in mental functioning)? 3. What can we learn about mood and sleep in MSA, through visualizing the serotonin system in the brain? How does this relate to symptoms that subjects report in these often underappreciated areas? To answer these and other questions, investigators will take images of specific nerves in the brain and heart using Positron Emission Tomography (PET) scans. Such imaging gives us information that cannot be obtained from MRIs and CT scans. We will measure the levels of several nerve cell types: serotonin, acetylcholine, and norepinephrine. Subjects will also have many standardized assessments including quality-of-life and symptom assessments, neurological examination, autonomic assessments, neuropsychological assessments, coordination tests, and even assessments of vision and sense of smell. By pooling these results from many MSA patients, and comparing with other diseases (such as Parkinson's disease) we hope to gain a better understanding of what is happening early in MSA. Such knowledge could be very valuable in future efforts to develop better therapies in this rare disease.

Conditions

Multiple System Atrophy - Parkinsonian Subtype (MSA-P), Multiple System Atrophy - Cerebellar Subtype (MSA-C)

Study of Rifampicin in Multiple System Atrophy
Description

The purpose of this study was to determine whether Rifampicin was effective in slowing or reversing the progression of multiple system atrophy (MSA). Research studies indicate that there is an abnormality in protein synthesis and structure in parts of the brain responsible for MSA (protein misfolding) and the drug Rifampicin could potentially prevent or reverse this protein alteration. The study was done on participants with early MSA. The study consisted of taking the drug 2 times a day for 12 months. Participants underwent an evaluation of symptoms and function and will underwent a neurologic examination at the beginning of the study, at 6 months and at 12 months. They were also be contacted at 3 and 9 months by telephone. Studies were done at 10 participating sites.

Conditions

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)
Description

To test the clinical effect of rasagiline on subjects with MSA of the parkinsonian subtype.

Conditions

Multiple System Atrophy

Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins
Description

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. The cause of the MSA remains unclear. Available treatment is symptomatic only and does not alter the course of disease. Although the cause of MSA remains unclear, there is evidence of presence of common neuroinflammatory mechanisms in the MSA brains including activation of microglia and production of toxic cytokines. This research protocol is based on hypothesis that the MSA progression can be altered by blocking the neuroinflammatory activity. This protocol includes administration of intravenous immunoglobulin (IVIg). IVIg contains antibodies derived from human plasma which can block the inflammatory responses in the brain that can lead to loss of brain cells.

Conditions

Multiple System Atrophy

Autologous suraL nervE Grafting to the Substantia nigrA in Patients With Synuclienopathies
Description

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 disease (PD) or Multiple System Atrophy (MSA). 7 participants will be enrolled, with 4 participants receiving the graft and 3 receiving a sham surgery. Eligible participants will be early in their diagnosis with a lower burden of symptoms. Participants will be followed initially for one year after surgery.

Conditions

Multiple System Atrophy, Parkinsons Disease

A Biomarker Study of ATH434 in Participants With MSA
Description

This study will assess the safety and efficacy of ATH434 in participants with a clinical diagnosis of Multiple System Atrophy

Conditions

Multiple System Atrophy

UB-312 in Patients With Synucleinopathies
Description

This is a Phase 1b study to determine the safety, tolerability, and immunogenicity of UB-312 in participants with multiple system atrophy (MSA), and in participants with Parkinson's disease (PD). UB-312 is a UBITh®-enhanced synthetic peptide-based vaccine and may provide an active immunotherapy option for treating synucleinopathies including the most prevalent form, PD; and the most rapidly progressive form, MSA.

Conditions

Multiple System Atrophy, Parkinson Disease

Effect of Verdiperstat on Microglial Activation in Well-characterized MSA Patients
Description

This study will comprise of two phases, an observational phase and a treatment phase. In the observational phase the specific aims are: 1. To determine the presence and regional distribution of microglial activation, as assessed by 18F-PBR06 PET, in subjects with MSA as compared to healthy controls, at baseline and at 6-9 months' follow-up. 2. To assess the relationship between microglial activation and clinical progression at baseline and follow-up. In the treatment phase the specific aims of the study are: The specific aims of the study are: 1. To assess whether verdiperstat (BHV-3241) reduces 18F-PBR06 PET signal, and thus microglial activation and inflammation, in well-characterized MSA patients. 2. To assess the relationship between PET changes and clinical progression at baseline and follow-up in patients treated with verdiperstat. 3. To assess the relationship between PET changes and volumetric brain MRI at baseline and follow-up in patients treated with verdiperstat. Currently there is no known disease modifying therapy for MSA. Recently, the drug verdiperstat (BHV-3241) has appeared in the investigational arena specifically for the indication of Multiple System Atrophy. Verdiperstat (BHV-3241) is currently being used in a phase 3 active drug trial at Massachusetts Hospital. Verdiperstat (BHV-3241) is known to target Myeloperoxidase, an enzyme implicated in neuroinflammation, a major driver in disease pathogenesis. Our previous study (IRB protocol #2016P002373) demonstrated that applying TSPO (translator protein) PET imaging enabled us to track changes in neuroinflammation and thus provide a viable biomarker for disease progression. In this pilot study, the investigators aim to assess the effect of an investigational drug, verdiperstat (BHV-3241) on microglial activation in MSA patients using \[F-18\]PBR06 and to link it with clinical and morphometric MRI brain changes following treatment.

Conditions

Multiple System Atrophy, Multiple System Atrophy, Cerebellar Variant, Multiple System Atrophy, Parkinson Variant (Disorder), Multiple System Atrophy (MSA) With Orthostatic Hypotension

Reduction in Splanchnic Capacitance Contributes to Sympathetically Dependent Hypertension in Autonomic
Description

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. Normally, the autonomic nervous system controls how much blood flows in the veins. In people with high blood pressure, however, the autonomic nervous system is changed. This change may affect the way blood flows in the veins of the abdomen which may play a role in their high blood pressure. About 32 participants will be screened for the study. The investigators estimate 16 will be eligible to participate in all of the study days.

Conditions

Multiple System Atrophy, Orthostatic Hypotension, Supine Hypertension

AZD3241 PET MSA Trial, Phase 2, Randomized,12 Week Safety and Tolerability Trial With PET in MSA Patients
Description

AZD3241 myeloperoxidase (MPO) inhibitor trial is assessing safety and tolerability, randomized trial, in patients with Multiple System Atrophy.

Conditions

Multiple System Atrophy, MSA