View clinical trials related to Huntington Disease.
Filter by:The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of PTC518 in participants with HD.
This is a single-center, randomized, double-blind, placebo-controlled, multiple-ascending doses trial to evaluate the safety, tolerability and pharmacokinetic of LPM3770164 sustained-release tablets orally administered in healthy subjects under fasting state, providing the rationale information for later clinical trials.
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate diverse disease research using cells from the body (such as skin or blood cells) to make stem cells and other types of cells, conduct research on the samples, perform genetic testing, and store the samples for future use. Through this research, researchers hope to identify future treatments or even cures for the major diseases of our time.
The primary objective of this study is to discover blood-based biomarker of brain Huntingtin (HTT) protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials. Secondary objectives of this study include developing more accurate biomarkers of Huntington disease (HD) progression or conversion and to develop standard practices for extracellular vesicle biomarker discovery research. The investigators hypothesize that brain-derived extracellular vesicles (EVs) isolated from human biofluids contain biological cargo specific to their tissue of origin that could allow their use as brain biomarkers for HD. EVs are lipid bilayer-delimited particles that are naturally released from cells in the brain. The investigators will investigate if EVs contents reflect the pathological alterations occurring with disease progression when compared with EVs isolated from biofluids of healthy non-HD persons.
This is an open label, dose escalation clinic trial of ER2001 intravenous injection to evaluate safety, tolerability and pharmacokinetics of ascending single and multiple doses of intravenously administered ER2001 in patients with early manifest Huntington's Disease. Furthermore, pharmacodynamics in particular target engagement, and early clinical signs of efficacy will be assessed. This study will evaluate increasing doses of ER2001 in sequential cohorts. ER2001 was escalated over 4 dose levels . The planned duration of this treatment is 14 weeks.
Time processing is fundamental to survival and goal reaching in humans. Different time scales (seconds, minutes, and beyond) are processed through specific cognitive processes involving different neural representations. It is generally agreed that time scale in seconds-to-minutes range named "interval timing" would be anatomically linked to the striatum. Indeed, it is possible to demonstrate a deficit of interval timing processes in patients suffering from striatal damage (Huntington's disease). However, recent findings show involvement of a second brain structure, the hippocampus, in interval timing processing in the minutes range, suggesting an interaction between the striatum and hippocampus. Presumably, patients with hippocampal damage (Alzheimer's disease) would specifically show a decrease in performance for this minutes-range time scale. This study aims to provide a better understanding of the role of the striatum in the treatment of time and its interactions with other brain structures such as the hippocampus. More specifically, it is unclear whether the striatum plays a platform role that would always be involved regardless of the time scale, as suggested by the unified model of time or whether different brain structures is solicited according to the time scale, as suggested by the modular system model. In order to elucidate these issues, a potential double dissociation between brain structures and time scales will be tested.
The survey and full study information can be accessed here: https://southampton.qualtrics.com/jfe/form/SV_8iEedDJZy4xaiI6 The goal of this anonymous survey is to benchmark integrated care from the perspective of service users in adults living with Huntington's Disease, including informal caregivers. The main question it aims to answer is: Is standard of care for people living with HD in England person-centred integrated care from the perspective of service users? Participants will complete a one-time anonymous survey either online or on paper, according to their preference, that takes an average of 20-30 minutes. Participants can take breaks as needed, picking up the survey from where it was paused.
The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington's disease. Another aim is to determine the concentrations of the study drug in the cerebral spinal fluid and blood after single and multiple doses. Study drug will be administered by lumbar intrathecal bolus injections.
OBJECTIVES: The primary study objective is to collect blood from participants with Huntington's Disease in order to validate a CE marked Cytosine, Adenine, Guanine (CAG) assay for use in future studies for Huntington's Disease. The secondary study objective is to create a biorepository that can be used to identify disease associated biomarkers and potential targets with immune and multi-omics profiling. The disease sample collection and analysis will be the foundation for an extensive network of biospecimen access and linked datasets for future translational research.
The goal of this observational study is to learn about brain development in Juvenile-onset Huntington's Disease (JoHD). The main questions it aims to answer are: - Is brain development different in JoHD than Adult-onset Huntington's Disease (AoHD)? - Can reliable biomarkers for JoHD be found in brain structure and function? Participants will be asked to complete cognitive tests, behavioral assessments, physical and neurologic evaluation, and MRI. Data collected will be compared to populations who are at-risk for HD and who have been diagnosed with HD as adults.