An Exploratory Study of the Potential for Rational Immune System Manipulation to Prevent Emergence of Synucleinopathy Manifestations in Persons with REM Sleep Behavior Disorder (RBD)

Date Added
September 3rd, 2026
PRO Number
Pro00151041
Researcher
Christine Cooper

List of Studies


Keywords
Parkinsons, Sleep Disorders
Summary

The purpose of this study is to determine if a drug called adalimumab can reduce or prevent emergence or progression of synuclein-related neurodegeneration, or nerve cell death, in people diagnosed with REM Sleep Behavior Disorder (RBD.) About 108 individuals, aged 50-80, will be enrolled in this study, half of whom will receive the active study drug and half of whom will receive placebo. Participants would receive 40 mg adalimumab, administered subcutaneously every 2 weeks or matching placebo for up to 2 years (96 weeks).

Institution
MUSC
Recruitment Contact
Sandra Wilson
8437924616
movementresearch@musc.edu

A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF VIM0423 IN INDIVIDUALS WITH ISOLATED DYSTONIA

Date Added
February 18th, 2026
PRO Number
Pro00149062
Researcher
Christine Cooper

List of Studies


Keywords
Movement Disorders
Summary

Dystonia is a movement disorder that causes muscles to contract and/or spasm. This may be painful and can affect the person's ability to complete daily tasks. Dystonia may affect one or multiple parts of the body. Botulinum toxins (BoNT) are the only approved drug in the United States to treat dystonia, and this is only for dystonia of the neck or the eye. There are currently no approved oral treatments for dystonia. Most current treatments only provide relief of symptoms.
The purpose of this study is to learn about the effects of the research drug (VIM0423), to find the best dose for treating dystonia, and to see how safe VIM0423 is for patients with dystonia.
This research study is studying VIM0423 as a possible treatment for dystonia. It is being developed to be a combination dose of: VMA-1001 given with VMA-1002.
• VMA-1001 and VMA-1002 will be taken in separate oral doses at the same time.
• VMA-1001 is an extended release (ER) modified version of trihexyphenidyl (THP).
• VMA-1002 is a formulation of bethanechol (BTC).
THP and BTC are medicines approved by the U.S. Food and Drug Administration (FDA); however, the Sponsor is investigating a different formulation of THP referred to as VMA-1001 and a different formulation of BTC referred to as VMA-1002. The purpose is to attempt to minimize some side effects of THP and is therefore considered an investigational drug in this study. An investigational use is one that is not approved by the FDA.
You may be in this study for up to 32 weeks from the time you consent until the last study visit.
You will be seen at the study site 6 times (Screening, Day 1, Day 30, Day 60, Day 95, and Day 125) and will complete 4 telephone calls (Day 6, Day 13, Day 20 and Day 105). You may be asked to come for extra visits at any time during the study if the study doctor decides that extra tests are needed for your safety.
Side effects associated with the study drug are dry mouth, dry eyes, blurred vision, dizziness, mild nausea and feeling nervous.
You do not need to take part in this study to receive treatment for your isolated dystonia. The study doctor will explain other options that are available to you. Your other choices may include treatment with other medicines for isolated dystonia, another investigational treatment, treatment that makes you feel more comfortable but will not have an effect on your isolated dystonia, or no treatment.

Institution
MUSC
Recruitment Contact
Robin Bulgarino
8437921115
bulgarino@musc.edu

AN OBSERVATIONAL STUDY OF INDIVIDUALS WITH ISOLATED DYSTONIA

Date Added
December 15th, 2025
PRO Number
Pro00148611
Researcher
Christine Cooper

List of Studies


Keywords
Movement Disorders
Summary

This is an observational study to further characterize and quantify disease severity
and impact (including change over time) in individuals with either segmental, multifocal, or generalized dystonia. Subjects will be aged 12-65 years old and clinically diagnosed with isolated (or primary) dystonia. Participation will include 2 visits over 120 days in which subjects will be asked to answer questions about their dystonia and related symptoms.

Institution
MUSC
Recruitment Contact
Sandra Wilson
8437924616
wilsosan@musc.edu

Neuron23, Inc. / "A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of NEU-411 in Companion Diagnostic-Positive Participants with Early Parkinson's Disease (NEULARK)"

Date Added
May 13th, 2025
PRO Number
Pro00142584
Researcher
Christine Cooper

List of Studies


Keywords
Parkinsons
Summary

The goal of the research study is to see if NEU-411 (1) will help prevent or slow the worsening of LRRK2 driven Parkinson's Disease (PD) and, (2) is safe. This study will also determine how your body processes NEU 411 and what NEU-411 does to your body. The study doctor will discuss with you whether you are eligible to be part of this study. As part of this study, you will receive either NEU-411 or placebo (inactive substance). The study drug is in tablet form and is taken by mouth. The study will last about 67 weeks. You will come in for 19 visits. In addition to in-person visits with the study team, you will be required to complete daily tests using a smartphone and occasionally be asked to wear a smartwatch continuously for 1-week periods. These technologies will be provided to you for use during the duration of the study and you will be trained on how to perform these activities. The NEU-411 is investigational and has not been approved by the US Food and Drug Administration as well as the investigational Companion Diagnostic (CDx).

Institution
MUSC
Recruitment Contact
Robin Bulgarino
8437921115
bulgarino@musc.edu



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