41 Clinical Trials for Various Conditions
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).
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.
The specific aims of the study are: Primary: To determine the presence and regional distribution of microglial activation, as assessed by \[F-18\]PBR06 PET, in subjects with MSA as compared to healthy controls, at baseline and at 9 months follow-up. Secondary: To assess the relationship between microglial activation and clinical progression at baseline and follow-up. Hypothesis: The working hypothesis is that there is microglial activation in Multiple System Atrophy and that the presence and regional distribution of microglial activation is different in MSA versus healthy controls and correlates with disease severity and comorbidities.
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.
A Phase 2a Study of Foralumab Nasal in Patients with Multiple System Atrophy (MSA)
This study will assess the safety and efficacy of ATH434 in participants with a clinical diagnosis of Multiple System Atrophy
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.
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.
AZD3241 myeloperoxidase (MPO) inhibitor trial is assessing safety and tolerability, randomized trial, in patients with Multiple System Atrophy.
Investigators hypothesize that there are specific characteristic of each cognitive and motor condition that can be defined using brains scans.
To test the clinical effect of rasagiline on subjects with MSA of the parkinsonian subtype.
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.
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.
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.
This study is an observational, prospective genetic study. It aims to obtain DNA for research and testing from patients with PSP, CBS, MSA, and related neurological conditions and their families. Up to 1,000 adults who have been clinically diagnosed with PSP, CBS, MSA, or related neurological conditions will be enrolled. The study intervention involves sequencing of participant blood samples using non-CLIA-approved whole genome sequencing at the National Institutes of Health. Pathogenic variants that are deemed possibly related to these conditions will be confirmed using CLIA-approved testing. The study involves minimal risk to participants.
A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of ampreloxetine in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects with Primary Autonomic Failure
This multiple-center, 3-part, single-blind dose escalation (Part A), randomized, double-blind (Part B), and open-label multiple dose extension (Part C) study will be conducted in male and female subjects with neurogenic orthostatic hypotension to evaluate the effect of TD-9855 in improving symptoms of orthostatic intolerance.
This study is designed to determine if magnetic resonance imaging (MRI) measures can be used to diagnose and monitor the progression of Parkinson's disease (PD) while distinguishing between PD and parkinsonisms \[conditions that are PD look-a-like diseases such as progressive supranuclear palsy (PSP) or multiple system atrophy (MSA)\] when combined with changes in certain proteins in body fluids that are related to iron (Fe).
An open, multi- center phase Ⅰ clinical study evaluating the safety and efficacy of autologous human polyclonal regulatory T cell injection (NP001 cell injection) in patients with Neurodegenerative diseases (ALS, MSA, AD).
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).
The purpose of this protocol is to create an active natural history cohort of patients with degenerative movement disorders, tracked in a clinical setting with clinical rating scales and neuroimaging. The overarching rationale is that neurodegenerative diseases may be heterogeneous, complex disorders. A new way of performing clinical trials in these patients may be in order and this protocol aims to build a longitudinally tracked clinical trial-ready cohort of patients. The purpose of this protocol is to establish an active natural history cohort of patients with neurodegenerative movement disorders who are deeply phenotyped and "clinical trial ready" across Mass General Brigham. After a thorough clinical diagnostic evaluation (this may include clinically indicated testing, for example MRI, FDG-PET, MIBG scan, polysomnography, genetic testing, autonomic function tests, inflammatory tests, skin biopsy) the investigators aim to achieve this through: 1. Longitudinal tracking of clinical progression through use of clinical scales including at the present time: UMSARS, BARS, MoCA and UPSIT, PROM, MDS-NMS, UPDRS, and SARA 2. Longitudinal tracking of disease progression through use of neuroimaging including at the present time: TSPO-PET and 3D MRI (see section 1.3) This is a pilot study designed to track patients with neurodegenerative movement disorders across Mass General Brigham through MRI and PET imaging modalities and clinical measures. Figure 5 represents the study design in detail. In short, subjects will be asked to visit Mass General Brigham every 6-9 months over the course of 18 months for imaging and clinical evaluation.
This is a single-arm, longitudinal, observational study on the use of wearable sensors and digital health technology to measure fall frequency and motor, speech, and cognitive function in patients with PSP over the course of approximately one year. Participants will perform supervised remote assessments monthly and in-person assessments approximately every 6 months.
The investigators aim to learn more about symptoms suggestive of a neurodegenerative process.
24 adults, between the ages of 18 and 75 years, with cerebellar ataxia will be enrolled in a 12 week trial of BHV-4157 for treatment of ataxia. BHV-4157 is a pro-drug of riluzole (which is currently FDA-approved for ALS, Lou Gehrig's disease). There will be 5 visits to UCLA required--Screening when general and neurological examination, blood and urine testing, ECG, and questionnaires will be administered; Baseline when general and neurological examination and questionnaires will be administered and study drug dispensed; Week 4 and Week 12 when general and neurological examination, blood and urine testing, ECG, and questionnaires will be administered; 2 weeks after finishing study drug when general examination and blood testing will be completed. There is an option for a 36 week extension of the study drug trial.
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 provide improvement in neurologic function for patients with certain neurologic conditions. http://mdstemcells.com/nest/
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 symptoms, and reduced rates of progression. After agreeing to participate, we will ask participants to fill our questionnaires about their experience with PD, their health in general, along with their food intake every six months for five years.
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.
The cohort registry is both retrospective and prospective, multicenter surveillance of subjects who underwent a prior hiatal hernia repair and Magnetic Sphincter Augmentation or fundoplication construction more than 2 years prior to initial study visit.
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.
The RETHINK REFLUX Registry is a post-market prospective, multi-center, observation, single arm, long-term safety surveillance registry of subjects implanted with the LINX device. The primary objective of the study is to confirm the long-term safety profile of the LINX device and procedure (implant/explant) up to 10 years post-implant.