View clinical trials related to Huntington Disease.
Filter by:The study is an open-label clinical trial evaluating whether music therapy combined with conventional management reduces irritability and impulsivity in 15 patients with early-stage Huntington's disease. This pilot study aims to show the interest of alternative non-pharmacological measures such as a digital music therapy tool, adapted to an audience of Huntington's patients, to help manage the psychobehavioral symptoms frequently observed in this affection, and to avoid breakdowns due to caregiver exhaustion.
The goal of this observational study is to learn about the first signs of disease in young adult carriers of the gene for Huntington's disease. The main questions to answer are: - what are the earliest signs of the disease? - can we identify the best time to intervene with treatment to prevent or delay onset of symptoms? - can we identify the most reliable markers of disease for use in prevention trials? Participants will undergo the following assessments: - clinical examination - cognitive and neuropsychiatric testing - brain imaging - biofluid sampling Researchers will compare gene carriers with matched controls to see if any of these measures show evidence of early disease effects.
The Project AWARE 2.0 Survey is intended to gain knowledge about the feelings, attitudes, and beliefs of HD family members, care partners and others affected by HD towards the way drug trials and observational studies are communicated and conducted.
This study will evaluate long-term safety and tolerability of valbenazine in participants with chorea associated with Huntington Disease (HD) who participated in Study NBI-98854-HD3006 (NCT04400331) in Canada.
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.
In this retrospective register study, clinically classified individuals with neurodegenerative disease from the years 2010-2021 will be verified from the clinical records from KUH and Oulu University Hospital (OUH). Based on the Finnish social security number, these individuals will be linked to the the national registers of Statistics Finland and Finnish Social and Health Data Permit Authority Findata including incomes, sociodemographic factors, education, occupation, criminal records as well as to the national registers including the bought pharmaceuticals, comorbidities and causes of death. For each study case, 10 randomly selected control cases, matched with age, sex and geographical area, will be used. The aim of the study is to examine: - 1) The prevalence of criminal and other disruptive behaviour in groups of different neurodegenerative diseases prior to and after the diagnosis - 2) Changes in employment, residency,income, and marital status prior to and after the neurodegenerative disease diagnosis - 3) Hospital diagnoses and reimbursable drugs prior to and after the diagnosis - 4) Causes of death in patients with neurodegenerative disease to study excess mortality of the patients
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.
Long-term follow-up of Huntington's disease patients treated with intrastriatal allografts is essential to assess the benefit/risk ratio of grafts as well as their effectiveness. Indeed, some patients are likely to develop adverse effects and the impact of alloimmunisation phenomena remains to be explored.
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting. The present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family.