All Huntington's Disease Clinical Trials

Background and study aims Huntington's disease (HD) is a condition that stops parts of the brain working properly over time. It's passed on (inherited) from a person's parents. It gets gradually worse over time and is usually fatal after a period of up to 20 years. The symptoms usually start at 30…

Background and study aims Huntington’s disease (HD) is an adult-onset genetic neurodegenerative condition, involving cognitive decline, motor impairments and emotional difficulties. Anxiety affects up to 71% of HD gene expansion carriers and damages quality of life, worsens other HD symptoms and…

This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).

Not provided at time of registration

Not provided at time of registration

NAC-preHD is a phase II randomized placebo controlled study of oral NAC among premanifest HD gene expansion carriers, with clinical and radiological outcome at three years.

Not provided at time of registration

The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.

Background and study aims Huntington’s disease (HD) is a brain disorder that is passed down from parent to child. In HD, cells in a part of the brain called the striatum are slowly lost. This leads to problems with movement, thinking and learning, which get worse over time. At the moment, there are…

Background and study aims The Sponsor is developing the test medicine zavacorilant (also known as CORT125329) for the potential treatment of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS - also known as Motor Neurone Disease), Huntington’s disease (HD), and Alzheimer’s…

Background and study aims Huntington’s disease (HD) is a rare, inherited disease that causes progressive degeneration of the nerve cells in the brain leading to gradual impairment in movement, learning abilities, and behaviour. Health authorities have not approved SAGE-718, the study drug for the…

A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease

Background and study aims: Huntington’s disease is a rare, inherited disease causing degeneration of the nerve cells in the brain, leading to gradual impairment in movement, learning abilities, and behavior. The DIMENSION Study is evaluating the safety and effects of an experimental oral drug…

This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 \[single-ascending dose\] followed by Part 2 \[open-label…

Background and study aims Huntington's disease (HD) is an inherited condition that causes damage to cells in the brain over time due to the production of an abnormal protein called mutant huntingtin (mHTT). Currently, there is no cure for HD and its progress cannot be reversed or slowed down. One…

The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet…

The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.

Background and study aims Huntington’s Disease (HD) is an inherited brain disease where people suffer from increasing problems with movement, thinking, and behavioural changes. At the moment there are no treatments that can slow down or stop the disease. Recent research studies have shown that…

Background and study aims Huntington’s Disease (HD) is a condition that causes problems with movement and thinking, which get worse over time. Many people with HD also experience depression, which affects their quality of life and ability to do everyday activities. Treating depression well could…

Huntington's disease (HD) is a hereditary neurodegenerative disorder characterized by progressively worsening motor, cognitive, psychiatric, and behavioral deficits. Cognitive deficits occur early on, affecting in particular executive functions (inhibition, flexibility), decision-making, memory…

Huntington's disease (HD) is a genetic, progressive neurodegenerative disorder characterized by early and widespread brain changes that can begin more than a decade before the onset of unequivocal motor signs. Although biological indicators of neurodegeneration-including striatal and cortical…

The purpose of the current proposal is to expand understanding of two currently available cognitive tools that are not typically used in Huntington Disease (HD) clinical trials that might be useful both for initial screening and for clinical trial application. One is the Coding Test and the other…

Not provided at time of registration

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…

MEND-HD is a longitudinal study evaluating the feasibility of passive monitoring of gait and chorea in patients with HD and the meaningfulness of these outcomes for patients with HD and their care partners/support persons. Participants will take part in four virtual visits with study investigators…

The goal of this clinical trial is to investigate if new, early biomarkers of the disease are available in presymptomatic genetic carriers of Huntington\'s diesase compared to healthy subjects. The main questions it aims to answer are: Does functional brain MRI is able to detect early…

Enroll-HD is a longitudinal, observational, multinational study that integrates two former Huntington's disease (HD) registries-REGISTRY in Europe, and COHORT in North America and Australasia-while also expanding to include sites in Latin America. More than 30,000 participants have now enrolled…

The proposed study is to evaluate the effectiveness of ExAblate Transcranial MRgFUS as a tool for creating a unilateral lesion in the Vim thalamus or the globus pallidus (GPi) in patients with treatment-refractory symptoms of movement disorders.

Background and study aims Huntington's disease (HD) is an inherited neurological disease, which over time results in progressive problems with movement, thinking and behaviour, and ultimately difficulties in undertaking the usual activities of daily living. It is known that keeping physically…

This study evaluates the feasibility and acceptability of using the artificial intelligence (AI) chatbot mobile application (an LLM-based Chatbot) to support the well-being of informal care partners of individuals with traumatic brain injury (TBI), dementia, or Huntington disease (HD). Over an…

Background and study aims Huntington’s Disease (HD) is an inherited disease that causes cell loss in brain regions important for learning and planning movements and doing two things at once (multitasking). HD leads to a worsening of these abilities. There is no cure for HD and there are currently…

FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records…

For participation in this epidemiological study, a single-day visit at the study site is required. Participants will be recruited from Huntington Disease clinics, and they will be asked to answer questions regarding their demographics, including sex, age, race and ethnicity, and their medical and…

The purpose of this study is to investigate whether speed-dependent measures of gait can be identified in patients with neurological conditions that affect gait, particularly in subjects with parkinsonian disorders.

The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement. Conducted at the Centre de Référence Liégeois des Maladies Neuromusculaires in Liège, Belgium, the…

Huntington's disease is a rare and fatal monogenic neurodegenerative disorder whose molecular origin is an expansion of CAG triplets within the first exon of the Huntingtin gene. Although a growing number of emerging therapies are in clinical trials, there are no proven neuroprotective or curative…

This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).

The purpose of this study is to find out if there is a connection between the naturally occurring bacteria in our bodies and the progression of Huntington disease. The investigators are trying to determine if patients who are diagnosed with adult-onset HD and who exhibit a rapid rate of disease…

Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder, characterized by movement disorders, behavioural disorders and cognitive decline. Especially the behavioural and cognitive symptoms of the disease lead to significant disability and burden for patients as well as…

The researchers hope to find out effects of transcranial direct current stimulation (tDCS) sessions on the behavioral symptoms of Huntington's Disease. If participants are eligible to continue, they will be provided a device to administer the tDCS for 30 minutes each day and be asked to answer…

The purpose of this study is to assess feasibility, acceptability, and safety of providing transcranial direct current stimulation( tDCS) to Huntingtons Disease (HD) patients in the early to middle stages and to assess the efficacy of tDCS for HD-related behavioral, cognitive and other symptoms

This is a FIH (first-in-human) study to evaluate the clinical utility of the radioligand \[11C\]CHDI-00491009 as a PET tracer that binds specifically to mutant huntingtin (mHTT) aggregates in Huntington's disease (HD). The study is divided into three cohorts defined by the Huntington's Disease…

iMarkHD is an adaptive, longitudinal positron emission tomography (PET) and magnetic resonance (MR) imaging study in Huntington's disease (HD) that aims to assess abnormal molecular, functional, and structural changes in participants' brains, ranging from several years before symptom onset to the…

Examine the effects of deutetrabenazine on functional speech and gait impairment

Intro Huntington's disease (HD) patients suffer from motor, cognitive and behavioral impairments, with heterogeneous phenotypes and variable time course. This leads to a high variance of HD markers, none of which is currently sensitive enough to 1) measure disease progression from small cohort…

Background and study aims People with neurodegenerative (relating to or characterized by degeneration of nervous tissue) conditions often have reduced breathing ability which impairs their ability to cough. Early findings from an ongoing study in people with Huntington’s disease (HD) show decreased…

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…

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 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…

AIM: to compare the sensitivity of SV2A PET and volumetric MRI to detect longitudinal striatal changes in premanifest HD. DESIGN: The investigators will include late premanifest HD mutations carriers and matched healthy controls. All subjects will undergo a clinical examination, with comprehensive…

This is a phase 1, single-center, single-dose, open-label mass-balance study to evaluate radioactive recovery rate, radioactive PK characteristics, metabolite identification, and to observe the safety in healthy male subjects of \[14C\] LPM3770164.

The purpose of this study is to assess preliminary efficacy of a tailored cognitive bias modification for interpretation (CBM-I) app for reducing anxiety in Huntington's disease and Parkinson's disease.

The objective of the study is to validate the use of wearable sensors and digital health technologies for monitoring disease activity in Huntington's Disease (HD). Healthy subjects, as well as subjects with documented diagnosis of HD will be screened and recruited at University of Rochester Medical…

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 purpose of this research study is to determine the optimal pulse repetition frequency of low-intensity focused ultrasound that is safe and effective in improving motor symptoms in patients with Huntington's disease.

The goal of this clinical trial is to improve communication among clinicians, patients with memory problems, and their family members. We are testing a way to help clinicians have better conversations to address patients' goals for their healthcare. To do this, we created a simple, short guide…

Not provided at time of registration

This study aims to develop and evaluate biomarkers using non-invasive optical coherence tomography (OCT) and OCT angiography (OCTA) as well as ultra-widefield (UWF) fundus photography to assess the structure and function of the retinal and choroidal microvasculature and structure in persons with…

This is the second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon continued demonstration of safety of AMT-130 administration. Cohort 3…

Not provided at time of registration

This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized…

The purpose of this research study is to determine whether an implantation of hNSC-01 is a safe and tolerable study intervention for Huntington's disease. This study is the first time that hNSC-01 is being tested in people.

Rare diseases (RDs) have been defined by the European Union (EU) as life-threatening or chronically debilitating conditions affecting less than 1 person in 2000. RDs are complex and often need special treatments, thus combined efforts are required to address them to improve diagnosis, care and…

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 goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and…

Background and study aims The Sponsor is developing a new recipe of the test medicine, branaplam, for the potential treatment of Huntington’s disease, a genetic condition that affects the nerves and how people move. The test medicine has been previously developed for a disease that affects nerves…

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…

The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet…

Background and study aims Huntington's disease (HD) is an inherited nervous-system-related disease, which over time causes problems with movement, thinking and behaviour and ultimately difficulties in undertaking usual activities of daily living. We know that keeping active (both physically and…

Background and study aims Huntington's disease (HD) leads to loss of mobility, with individuals eventually requiring assistance with all activities of daily living. Our research has provided evidence that people with early stage HD can benefit from a physiotherapy-led home-based exercise programme…

The goal of this clinical trial is to learn if oral supplement of nicotinamide riboside (NR), a form of vitamin B3, slows disease progression in adults with Huntington's disease. It will also learn about the safety of nicotinamide riboside. The main questions it aims to answer are: * Does NR slow…

Huntington's disease (HD) is a neurodegenerative pathology characterized by choreic hyperkinesias which represent the typical motor symptom and are represented by involuntary, aimless, irregular, recurrent, unpredictable and non-rhythmic movements of the trunk, face and limbs. Non-invasive brain…

Evaluate the safety and tolerability of combined oral thiamine with biotin therapy in patients with Huntington´s disease in mild to moderate stages and it is intended to evaluate the biological effect of the treatment in the central nervous system of these patients using as the main biomarker the…

Millions of persons are affected by brain diseases. The CereBank will be used to support research studies aimed at improving the diagnosis and treatment of brain diseases. Brain diseases can be inherited; developed due to diseases or aging; or can be due to trauma. The Central Florida does not have…