This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of valbenazine in subjects with Huntington's Disease (HD). The study includes a 4-week screening period, an 8-week dose-adjustment period, a 4-week maintenance period, and a final study visit 2 weeks following the final dose of study drug. Approximately 120 adult male and female subjects with motor manifest HD (a type of HD that causes uncontrolled movements and balance issues) and a genetic diagnosis of HD will be enrolled.
Study drug will be self-administered (in the presence of the subject's caregiver, if applicable) once daily (qd), preferably in the morning but before 1800 hours, beginning on Day 1. Study drug should be administered at approximately the same time each day during the study. Valbenazine will be supplied as orally administered capsules containing 20 or 40 mg of valbenazine. Subjects must swallow the capsules with approximately 4 ounces of water or other liquid, with or without food.
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.
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.
Hand disability after stroke has a profound negative impact on functional ability and independence. Hand therapy may be augmented with sensory stimulation for better outcomes. We have developed a novel sensory stimulation - unfelt vibration applied via a wristwatch. In this study, we will determine if combining this stimulation with hand task practice is superior to hand task practice alone.
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.
Transcranial direct current stimulation (tDCS) has shown the potential to improve symptoms in patients with motor deficits, however its effects have not been consistent in randomized studies to date, limiting widespread adoption of this technology. A critical gap in our knowledge is a detailed understanding of how tDCS affects motor areas in the brain. We propose using tDCS while recording directly from motor cortex using subdural electrocorticography (sECoG) in patients undergoing deep brain stimulation surgery. We expect this novel approach to broaden our understanding of tDCS application and possibly lead to therapeutic advances in this population.
The primary objective of Enroll-HD is to develop a comprehensive repository of prospective and systematically collected clinical research data (demography, clinical features, family history, genetic characteristics) and biological specimens (blood) from individuals with manifest HD, unaffected individuals known to carry the HD mutation or at risk of carrying the HD mutation, and control research participants (e.g., spouses, siblings or offspring of HD mutation carriers known not to carry the HD mutation). Enroll-HD is conceived as a broad-based and long-term project to maximize the efficiencies of non-clinical research and participation in clinical research while ensuring privacy and protections for consenting research participants.
Over many years, we have learnt that the brain's connections with the spinal cord change in response to injury or training. Because brain-spinal cord (i.e., corticospinal) pathways are very important in movement control, restoring function of these pathways could help to restore useful movement after spinal cord injury (SCI). In this project, we hypothesize that operant conditioning training of the muscle response to non-invasive transcranial magnetic stimulation can strengthen the functional connectivity of corticospinal pathways and thereby alleviate movement problems in people with chronic incomplete SCI. Specifically, through this project, we will investigate the effects of strengthening the corticospinal connection to the ankle dorsiflexor muscles through operant up-conditioning of the muscle evoked response, in hope to enhance the function of corticospinal pathways and alleviate foot drop (i.e., weak ankle dorsiflexion resulting in toe drop and drag) during walking in people with chronic incomplete SCI.
Spinal reflexes take important part in our movement. After spinal cord injury (SCI), reflexes often change. For many years, researchers and doctors have assumed that abnormally acting spinal reflexes lead to movement problems, without clear scientific evidence. For example, in people who suffer spasticity, a common problem after SCI, walking is disturbed, presumably because stretch reflexes (e.g., knee jerk reflex) and some other reflexes are not working well. Yet, which reflex is causing a problem in what way has not been well understood. Such understanding is very important in developing and applying effective therapies for improving gait recovery after SCI. Therefore, in this project, we are studying spinal stretch reflexes and other reflexes during walking, to understand how these reflexes contribute to spastic gait problems in people with chronic incomplete SCI. Successful completion of this project will result in better understanding of spastic gait problems, which in turn, will help us develop more effective therapy application and improve the quality of life in people after SCI.
This is an exploratory study and the information obtained may lead to new findings regarding the inflammatory and neurodegenerative mechanisms in the progression of PD and help to develop new drugs to halt the disease progression. The study simply involves a one time blood draw.