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.
This study will test if bemdaneprocel (the "study drug") is safe and effective in improving symptoms in participants with Parkinson's Disease. The symptoms of PD are due to a loss of cells that make a specific chemical in your brain called dopamine. The study drug is a cell therapy that contains live brain cells that produce dopamine. This study will last 5 to 8 years, and depending on randomization group between 23-32 study visits.
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.
The purpose of this research study is to examine the effect of a brain stimulation training to improve the function of brain-spinal cord- muscle connections.Because brain-to-muscle pathways are very important in our movement control, restoring function of these pathways may improve movement problems after injuries. Spinal cord injury causes damage to the brain-to-muscle connection. However, when the injury is "incomplete", there is a possibility that some of the brain-to-muscle pathways are still connected and may be trained to improve movement function. For examining brain-to-muscle pathways, we use a transcranial magnetic stimulator. We hope that the results of this research study will help us develop new treatments for people who have movement disabilities. This study will require about 42 visits over the first 14 weeks, and another 6 visits over an additional 3 months. Each visit will take about 1 ½ hours.
Adherence to home exercise is important to achieve upper limb recovery after stroke. However, adherence is typically low. Therefore, a new home exercise program with an Apple Watch and iPhone app was created to improve adherence to upper limb exercises for stroke survivors at home. Participants will come to our lab to experience the new home exercise program. Participants who opt for home use will bring the device home to try the new home exercise program at home. The purpose of this study is for researchers to examine usability and feasibility of participants using the new home exercise program.
For this study, we are recruiting 54 individuals with Parkinson's Disease and Freezing of Gait (FOG) who are planning to undergo Deep Brain Stimulation (DBS). The objective of this study is to better understand the FOG response to DBS. Prior to DBS study participants will undergo a MRI scan, behavioral assessment related to walking, a cognitive evaluation, and assessment of other Parkinson's disease symptoms. Following DBS participants will repeat these assessments at multiple timepoints over the period of one year. Overall, participants will complete a total of 7 visits over a period of approximately 1 year.
The purpose of this study is to develop and test if upper limb task practice and muscle activity training improve upper limb function in stroke survivors. Participants will be asked to come to the laboratory 3 times a week for 6 weeks to receive upper limb task practice and/or muscle activity training. Participants will also come to the laboratory for additional 3 visits for assessments of upper extremity function. The total duration of the study will be 2.5 months.
The purpose of this research study is to identify brain activation patterns in response to deep brain stimulation (DBS). To participate in this study, participants are required to have had a DBS implant or to be healthy controls without a history of a neurological disorder. Participants will undergo screening, a motor assessment session and a 30-minute Magnetic Resonance Imaging (MRI) session where their DBS device will be turned on and off in a cycled pattern. This study will be separated over 3 visits (Visit 1 for screening, Visit 2 for motor assessments and Visit 3 for the MRI scans). The total study duration will be approximately 5 hours.
The purpose of the first portion of this study is to gather feedback from clinicians on the usability of the current system and procedure, so the researchers can make reflex training more useful and usable for improving recovery after spinal cord injury or other nervous system injuries and diseases. The researchers are recruiting 20 therapists who have been actively practicing physical medicine and 30 adults with no known neurological conditions to test system usability and the reflex operant conditioning protocol. For this portion of the study, there are 5 visits. We will also recruit 15 adults with no neurological injuries, 15 adults with neuropathic pain, and 15 adults with non-neuropathic pain to participate in one visit to provide feedback on sensation caused by stimulating electrodes.
The purpose of the second part of the study is to validate the capacity of the system to change the size of the targeted reflex. For this the researchers are recruiting 25 individuals with chronic incomplete SCI who have spasticity in the leg to participate in the reflex training procedure. The study involves approximately 45 visits with a total study duration of about 6 months.
Walking after a lower extremity amputation is often difficult. It is important that researchers and clinicians understand the mechanisms that inhibit normal walking function. In this study, we are recruiting individuals with lower extremity limb loss for a walking and balance investigation. We will also be studying matched healthy controls to do similar study procedures. All study procedures will occur on the campus of MUSC by a licensed Physical Therapist and experienced researcher. Any questions should be directed to the coordinator listed.