Clinical Trials for Huntington's Disease

Source: ClinicalTrials.gov — U.S. National Library of Medicine — A database of privately and publicly funded clinical studies conducted around the world (U.S. National Library of Medicine, public domain).

This page lists clinical trials related to Huntington’s Disease from the ClinicalTrials.gov database.

Trial Summary

• Total studies: 50
• Recruiting: 22
• Active, not recruiting: 10
• Completed: 10
• Other: 8

Results are refreshed periodically and may not reflect the most current information available from ClinicalTrials.gov.

Recruiting Trials

NCT02855476 — HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington’s Disease

HDClarity will seek at least 2500 research participants at different stages of Huntington’s disease (HD). The primary objective is to collect a high quality CSF sample for evaluation of biomarkers and pathways that will enable the development of novel treatments for HD. The secondary objective is to generate a high quality plasma sample collection matching the CSF collections, which will also b…

NCT03233646 — Retinal Imaging in Neurodegenerative Disease

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 mild cognitive impairment (MCI) and Alzheimer’s Disease (AD), Parkinson’s Disease (PD), or other neu…

NCT04012411 — Study of BDNF Pathway Biomarkers in the Cerebrospinal Fluid in Patients With Huntington’s Disease

Huntington disease (HD, 1.3/10 000) is an autosomal dominant disease due to an abnormal expansion of CAG triplets in HTT gene.

Several pathophysiological mechanisms have been evoked, including an alteration of the signaling pathway of the Brain Derived Neurotrophic Factor (BDNF), a neurotrophic factor involved in the survival of neurons (striatal and hippocampal) and synaptic plasticity. BDNF …

NCT05326451 — Home-based Transcranial Direct Current Stimulation Open Trial for Behavioral and Cognitive Symptoms in Huntington’s Disease

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

NCT06667414 — Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers

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 medication history. At the end of the visit, a blood sample will be drawn to allow testing with a s…

NCT06585449 — A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington’s Disease

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.

NCT06414967 — Study to Evaluate Music Therapy on Irritability and Impulsivity in Patients With Huntington’s Disease (MUSIC-HD)

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 …

NCT06147414 — Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

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 a…

NCT06826612 — A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington’s Disease

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

NCT06546488 — Cognitive Assessment Tools for Huntington’s Disease.

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 is the Self-Administered Gerocognitive Examination (SAGE). Both the Coding Test and the SAGE have b…

NCT07010705 — Digital Measures for Clinical Trial Endpoints in Huntington’s Disease

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 to answer survey questions on movement and cognition, perform in-home movement assessments, and t…

NCT06807892 — Comparison of Two Cognitive-Motor Rehabilitation Approaches Via Exergames: a Study of Cognitive, Motor and Behavioral Functions in Huntington’s Disease Patients

Huntington’s disease (HD) is a rare, hereditary neurodegenerative disorder. It generally manifests itself between the ages of 40 and 50, and results in motor impairment (choreic movements, balance disorders, gait disorders, etc.), cognitive impairment (executive functions, attention, etc.) and behavioral impairment (apathy, depression, irritability, etc.). To date, there is no curative treatmen…

NCT06774443 — Hinting Task for Huntington’s 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 caregivers. One of the cognitive domains affected by HD is social cognition. Social cognition is the abil…

NCT06843252 — Home-based TDCS (Transcranial Direct Current Stimulation) for Cognitive and Behavioral Symptoms in Huntington’s Disease

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 questions with the study staff . Participants will be asked to return to the study center for follow u…

NCT07246941 — A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington’s Disease

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 extension (OLE)].

NCT07253038 — Evaluation of Three Tests to Assess Social Cognition in Huntington Disease

The goal of this observational study is to learn about the usefulness of a test of social functioning in persons with Huntington disease. Huntington disease affects motor function, psychological well-being and cognitive functions (“thinking abilities” such as paying attention, remembering and solving problems). It is also believed to affect important social functions, including the ability to u…

NCT07378644 — Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington’s Disease

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 certain requirements for physical ability and independence.

NCT06634628 — iMagemHTT-009- FIH Evaluation of Novel Mutant Huntingtin PET Radioligand [11C]CHDI-00491009

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 Integrated Staging System (HD-ISS): Cohort 1 - initial tracer validation (3 healthy controls (HCs)); …

NCT07326709 — A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington’s Disease

The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington’s disease (HD) compared to the control arm.

HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.

NCT07451613 — Safety and Tolerability Study of Human Neural Stem Cells for Huntington’s Disease

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.

NCT07513844 — Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation in Huntington’s Disease

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.

NCT07536061 — A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington’s Disease

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

Active, Not Recruiting

NCT02994719 — Gait Analysis in Neurological Disease

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

NCT05707663 — Longitudinal Assessment of Brain Structure and Function in Juvenile-onset Huntington’s Disease

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 as…

NCT07503743 — Biology-Driven Cognitive Profiling in Huntington’s Disease

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 atrophy, white-matter disruption, and elevated plasma and CSF biomarkers-are detectable during the prem…

NCT04713982 — Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease

Examine the effects of deutetrabenazine on functional speech and gait impairment

NCT05541627 — A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington’s Disease

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

NCT05686551 — GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington’s Disease

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).

NCT05596760 — Promoting Goals-of-Care Discussions for Patients With Memory Problems and Their Caregivers

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 called the “Jumpstart Guide.” The goal of this research study is to show that using this kind of guide…

NCT06626412 — Longitudinal SV2A PET and MRI in Premanifest HD

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 assessment of motor and non-motor symptoms, and imaging evaluation consisting of 18F-SynVesT-1 PE…

NCT07339514 — A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington’s Disease.

This is a dose escalation and expansion clinical study to evaluate the safety, tolerability, PK profile and preliminary efficacy of ER2001 Injection vs. placebo in subjects with definitive diagnosis of early manifest HD.

The study consists of a dose escalation phase (Part A, an open-label without placebo, which will be carried out firstly) and a dose expansion phase (Part B,randomized, blinded…

NCT06873334 — Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington’s Disease

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 certain requirements for physical ability and independence.

Not Yet Recruiting

NCT07315984 — Multi-Modal Digital Monitoring of Disease Symptoms Huntington’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 Center and Vanderbilt University Medical Center to participate in this 12-month observational stu…

NCT07409597 — Autobiographical Memory, Future Thought, and Eye Movements in Huntington’s Disease

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, attention (selective, sustained), perceptual and visuospatial skills, and information processing spe…

NCT07516899 — Mass Balance Study of [14C] LPM3770164 in Healthy Participants

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.

NCT07609108 — Pridopidine Phase 3 Study in Huntington’s Disease

The goal of this clinical trial is to learn if pridopidine can slow the clinical decline of Huntington’s Disease (HD) in adult participants. It will also inform about the safety of pridopidine.

The main questions the study aims to answer are:

Does pridopidine slow the overall worsening of HD over 1 year? Does pridopidine slow the worsening of specific aspects of HD over 1 year, namely the cli…

NCT07280650 — Feasibility and Acceptability of an LLM-based Chatbot for Family Caregivers: Evaluation Study

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 8-week intervention period, up to 60 care partners will engage with the application (app), spending fo…

Enrolling by Invitation

NCT06312189 — Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in Canada

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.

NCT07537075 — An Extension of SKY-0515 in Participants With Huntington’s Disease

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 certain requirements for physical ability and independence.

Completed Trials

NCT01860339 — Child to Adult Neurodevelopment in Gene Expanded Huntington’s Disease

Huntington’s Disease (HD) is an autosomal dominant disease manifested in a triad of cognitive, psychiatric, and motor signs and symptoms. HD is caused by a triplet repeat (CAG)expansion in the gene Huntingtin (HTT). This disease has classically been conceptualized as a neurodegenerative disease. However, recent evidence suggests that abnormal brain development may play an important role in the …

NCT00465790 — Research of Biomarkers in Parkinson Disease

The main goal of the GENEPARK consortium is to employ innovative haemogenomic approaches to determine gene expression profiles specific for genetic and idiopathic Parkinson’s disease (PD) patients. These gene expression signatures will be utilised clinically as non-invasive diagnostic tests for PD. The sensitivity of the newly developed diagnostic test will be determined by extensive validation…

NCT01842919 — Dance and Huntington Disease

In this project investigators will evaluate the benefits of contemporary dance training using a comprehensive test battery combining standard neuropsychological batteries, psychological questionnaires on emotion, empathy and quality of life, structural magnet-resonance tomography (MRI), as well as psychophysical tests on movement recognition and agency, the sense of being in control of one’s ow…

NCT01882062 — Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease

The purpose of this project is to study the efficacy of an anaplerotic treatment on brain energy profile evolution at an early stage of the Huntington disease.

NCT02453061 — A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington’s Disease

In the study the investigators plan to include 100 early affected HD patients (5 ≤ UHDRS ≤ 40) in a randomized, double-blind, controlled study in 2 centers (France and the Netherlands). Patients will receive either triheptanoin at 1g/kg of body weight per day (n = 50), or a control oil (n = 50) at 1g/kg of body weight per day for 6 months followed by an open label phase using triheptanoin for a…

NCT05157074 — Group Drum-Based Music Therapy Intervention for Parkinson’s Disease/Huntington’s Disease

Participants in this study (18-89 years) with Parkinson’s disease or Huntington’s disease receive drum classes twice a week for 12 weeks (24 lessons). All participants also participate in study visits for assessments before the beginning of the study, at the 6 week mark, at the 12 week mark and at the 18 week mark so that the investigators can assess the short and long term effects of drum clas…

NCT04400331 — Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease

This is a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine, and to provide participants continued access to valbenazine for the treatment of chorea associated with Huntington disease.

NCT05358717 — A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington’s Disease (HD)

The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.

NCT07601516 — Real World Effectiveness and Safety of Deutetrabenazine in Adult Chinese Patients With Huntington’s Disease (HD) Chorea in China

The Primary Objective: To evaluate the real-world effectiveness of deutetrabenazine in adult patients with chorea associated with Huntington’s disease in China.

The Secondary Objectives: To evaluate the real-world safety of deutetrabenazine in adult patients with chorea associated with Huntington’s disease in China.

NCT07344519 — The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington’s Disease.

This is an open label, dose escalation clinic trial to evaluate safety, tolerability and pharmacokinetics with ER2001 Intravenous Injection repetitive treatment in Huntington’s Disease patients who Completed Study ER2001-001（NCT06024265）. Furthermore, pharmacodynamics in particular target engagement, and clinical signs of efficacy will be assessed. This study will evaluate increasing doses of E…

Other (Withdrawn)

NCT07299682 — Facilitating Effective Eating With DoddleBags: A Single-Arm Acceptability Study Of DoddleBags to Assess Impact on Self Feeding Behaviours in a Huntington’s Disease Population

Primary Objectives

• To assess the acceptability of using the DoddleBags feeding aid in people with HD, by using a food diary to track food intake and using semi-structured interviews to ask questions about how easy or difficult it was to use a DoddleBag for each meal compared to not using one, and learn more about the impact of using DoddleBags on self-feeding behaviours.
• To review the impa…