HD Clinical Trials and Studies
May 09, 2022
HDYO has more information about HD available for young people, parents and professionals on our site:
www.hdyo.org
Where to Find Trials
HD TrialFinder is a clinical trials matching service: a way for individuals with Huntington’s disease, caregivers, healthy volunteers, and physicians to connect with current research studies. It includes an easy-to-use website and free call center staffed by trained HD clinical trial navigators. People with HD, care partners, and healthy volunteers are all needed today to participate in different aspects of HD research.
Visit HDSA TrialFinder Visit EHA TrialFinder
ClinicalTrials.gov is a website that lists research studies from around the world. This site can be challenging for the community to navigate if they don't have a scientific background. We suggest starting with the different TrialFinder sites first or review the resources below.
Summary of Current HD Research
Research into Huntington’s disease is an incredibly active field. This page summarises some of the major research studies currently underway that pertain to young people that HDYO serve. We’ve split the studies up into ‘observational’ and ‘interventional’ research. Observational research collects information about participants without interfering. This is very important to learn about a disease, find out how it usually progresses and discover measures like biomarkers that can be used for interventional research. Interventional research tests treatments for a disease, an important step in developing new drugs. For a simple explanation of the drug development process, see our video here.
This list will be updated as new information is made available. If you are involved in a HD research project not listed and would like it featured on this page, please get in touch by emailing rebecca@hdyo.org.
Observational Research
HDYO Survey Series
Who runs this study? Huntington's Disease Youth Organization
What’s its current status? Active.
Where is it running? Internationally.
Who’s potentially eligible? HD community members at least 18 years and older.
What are they investigating? HDYO is hosting a series of surveys to better understand behavior, stigmas, awareness and education around several areas of individuals impacted by Huntington's disease.
ChANGE HD
Who runs this study? The University of Iowa
What’s its current status? Active and recruiting.
Where is it running? United States.
Who’s potentially eligible? Young people aged 6 to 30 who are at risk for HD.
What are they investigating? ChANGE HD is a brain imaging study which will help explain how the gene responsible for HD affects brain development, and hopefully find a way to identify the best time for gene therapy during development.
Enroll-HD
Who runs this study? The CHDI Foundation
What’s its current status? Active and recruiting.
Where is it running? Argentina, Australia, Austria, Belgium, Canada, Chile, Colombia, Czech Republic, Denmark, France, Germany, Ireland, Italy, Netherlands, New Zealand, Poland, Portugal, Russia, Spain, Switzerland, United Kingdom, United States.
Who’s potentially eligible? Anyone from a HD family, aged 18+, and children under 18 with a diagnosis of juvenile HD.
What are they investigating? Enroll-HD was launched in 2012 and has over 20,000 active participants. The goals of Enroll-HD include to better understand HD, to improve the design of clinical trials and to improve clinical care for HD patients. Participants visit their local study site annually to undergo assessments and give biospecimens.
FOCUS Online
Who runs this study? The CHDI Foundation
What’s its current status? Active
Where is it running? International and online
Who’s potentially eligible? Individuals who are gene positive for HD, 18 or older, and can speak English
What are they investigating? The purpose of the Study is to determine whether the FuRST 2.0 Scale can reliably and accurately capture information about the functional abilities of people with HD.
HDClarity
Who runs this study? University College London and the CHDI Foundation
What’s its current status? Active and recruiting.
Where is it running? Canada, Germany, Italy, Poland, Spain, United Kingdom, United States.
Who’s potentially eligible? Adults aged from 21 to 75, who fall under one of the following categories: healthy control, early pre-manifest, late pre-manifest, early manifest, moderate manifest, late manifest.
What are they investigating? HDClarity is a study designed to collect cerebrospinal fluid, the fluid that surrounds the brain and spinal cord. The samples and information collected will be used to study HD, including identifying and evaluating biomarkers, which may help researchers better understand the disease. In the future, these biomarkers could be used to help design clinical trials of new treatments.
HD-JUNIOR
Who runs this study? Leiden University Medical Center
What’s its current status? Active and recruiting.
Where is it running? The Netherlands.
Who’s potentially eligible? People in the Netherlands who developed HD symptoms before the age of 21.
What are they investigating? HD-JUNIOR is a national registry for patients with JoHD. They will collect medical information from participants' doctors and contact information so they can get in touch about future research opportunities. There is also an optional questionnaire that the participant or caretaker can fill in.
HD-YAS 1.0
Who runs this study? University College London Huntington’s Disease Centre
What’s its current status? Completed
Where is it running? UK
Who’s potentially eligible? Adults between the ages of 18 and 40 years old who are at-risk and premanifest (show no clinical symptoms of HD), including those who carry the HD gene or those who do not
What are they investigating? HD-YAS 1.0 studied a cohort of young adult HD gene expansion carriers decades before expected symptom onset to characterize the very earliest signs of disease-related brain changes and identify whether there is any identifiable early functional impairment. HD-YAS 1.0 showed that, although function was completely normal, there was some evidence of subtle early changes in gene carriers decades from developing the disease.
HD-YAS 2.0
Who runs this study? University College London Huntington’s Disease Centre
What’s its current status? Active
Where is it running? UK
Who’s potentially eligible? Adults between the ages of 18 and 40 years old who are at-risk and premanifest (show no clinical symptoms of HD), including those who carry the HD gene or those who do not
What are they investigating? The purpose of HD-YAS 2.0 is to determine whether there is any change over time in adults ages of 18-40 years old who are premanifest (show no clinical symptoms of HD) at enrollment in the study. The 131 participants who took part in HD-YAS 1.0 are being invited back to undergo similar assessments so we can look at what has changed. In addition, new participants are also being included to account for participants from the first study who may not want to participate again. This will help us to guide the use of any potential future treatments, so that they can be given at the earliest and most effective time in order to prevent or treat HD disease progression.
JOIN-HD
Who runs this study? HDYO
What’s its current status? Active and recruiting.
Where is it running? Worldwide.
Who’s potentially eligible? People with symptoms consistent with JoHD, caregivers of someone with symptoms of JoHD and previous caregivers of someone who had JoHD.
What are they investigating? JOIN-HD is an international registry where participants self-enrol and answer questionnaires online. The project aims to create a global community of families impacted by JoHD, to increase knowledge of this disease, and to facilitate future research.
Kids-JHD
Who runs this study? The University of Iowa
What’s its current status? Active and recruiting.
Where is it running? United States.
Who’s potentially eligible? Young people aged 4 to 30 years old with a diagnosis of JoHD.
What are they investigating? This study is trying to identify and measure common symptoms and developmental patterns that might be happening in JoHD.
MyHD Story
Who runs this study? The Huntington Study Group
What’s its current status? Active and recruiting.
Where is it running? United States with hopes of including international after pilot study is complete.
Who’s potentially eligible? * Adults age 18 years and older * Willing and able to provide informed consent electronically. * Individuals answering as a participant must self-report they have been diagnosed with HD by a doctor. * Ability to answer online questions or direct someone else to enter answers for them. * Ability to ambulate independently and take care of some of his or her personal needs. * Ability to read and understand English. * Willing to create a unique identifier based on personal demographic information. * Residing in the United States or its territories * Owning or having access to an electronic device (laptop, smartphone, or computer) and secure internet connectivity
What are they investigating? This study will help to understand how Huntington disease (HD) affects patients, care partners, and those at genetic risk for HD.
PREVENT-HD
Who runs this study? University of Wisconsin, Madison
What’s its current status? Active and recruiting.
Where is it running? United States.
Who’s potentially eligible? Adults from HD families who carry or do not carry the HD gene, people who have recently been diagnosed with HD and are in the very early stages of the disease, and people who are gene-positive, but are not currently showing any symptoms.
What are they investigating? This study aims to build a case for testing treatments much earlier in people at risk for HD. Starting treatment sooner may help delay the start of symptoms or slow down the progression of the disease. This study wants to plan for future clinical trials by finding and tracking subtle but measurable changes in behaviour, cognition, and emotional responses that occur before the more visible HD symptoms appear.
SHIELD HD
Who runs this study? Triplet Therapeutics
What’s its current status? Active, not currently recruiting.
Where is it running? Canada, France, Germany, United Kingdom, United States.
Who’s potentially eligible? Adults aged 18 to 63 with pre-manifest or early manifest HD.
What are they investigating? SHIELD HD is an international study to assess the natural history of HD and its biomarkers associated with modulation of the number of CAG repeats in the mutant huntingtin gene. The results of this study will inform assessments for a future interventional trial.
To what extent can an AI Character offer peer support, motivation, self-efficacy and knowledge transfer for Preventive Neurology
Who runs this study? Experience & Empathy Lab [fEEL] and The Big Anxiety Research Centre at the University of New South Wales (UNSW) in Australia
What’s its current status? Active
Where is it running? Global
Who’s potentially eligible? Adults aged 18-50, part of the Huntington’s Disease community, interested in brain health, ability to speak English
What are they investigating? Are you interested in exploring the potential of AI technology to enhance your brain health journey? We invite you to participate in our research study which aims to explore to what extent an AI character, developed from lived experience, can provide peer support, enhance self-efficacy and motivation, and facilitating knowledge exchange for individuals who may be ambivalent, unaware, or unmotivated to engage with Preventive Neurology (brain health optimization).We're looking for participants aged 18-50 who are passionate about brain health, have access to a computer, and are fluent in English. Your involvement will include approximately 55 minutes to complete a survey, engage in a Microsoft Teams call facilitated exchange with an Ai Character, and participate in an informal interview. The attached Participant Information Statement, which contains a full description of the research activities, risks associated with these activities and any discomforts that you may experience during the research.
Interventional Research
Phase I study of ER2001 in Early HD
Who runs this study? ExoRNA Bioscience
What’s its current status? Active and recruiting.
Where is it running? China
Who’s potentially eligible? Adults between the ages of 25 and 55 with early manifest HD
What are they investigating? This clinical trial will assess the safety, tolerability, and pharmacokinetics of increasing doses of intravenously administered ER2001 in patients with early manifest HD. The study is expected to enroll 15 participants and run in Guangdong between April 2023 and October 2024.
DIMENSION
Who runs this study? Sage Therapeutics
What’s its current status? Active and recruiting.
Where is it running? United States.
Who’s potentially eligible? Adults aged 25 to 65 with pre-manifest or early manifest HD.
What are they investigating? This phase II trial will investigate the safety of the oral drug SAGE-718 and its effect on cognitive symptoms in participants with pre-manifest or early manifest HD.
KINECT-HD
Who runs this study? Neurocrine Biosciences and the Huntington Study Group
What’s its current status? Completed
Where is it running? Canada and United States.
Who’s potentially eligible? Adults aged 18 to 75 with a diagnosis of motor manifest HD.
What are they investigating? KINECT-HD conducted a Phase 3, randomized, double-blind, placebo-controlled study to investigate valbenazine (whose trade name is INGREZZA®) for the treatment of chorea in HD. Results demonstrated improvements chorea severity, compared to placebo and recently supported the ability of the U.S. Food and Drug Administration (FDA) to approved INGREZZA® (valbenazine) capsules for the treatment of adults with chorea associated with HD.
KINECT-HD2
Who runs this study? Neurocrine Biosciences and the Huntington Study Group
What’s its current status? Active and recruiting.
Where is it running? Canada and United States.
Who’s potentially eligible? Adults aged 18 to 75 with a diagnosis of motor manifest HD.
What are they investigating? KINECT-HD2 is an open-label rollover study of the previous trial KINECT-HD. This trial is investigating Valbenazine (whose trade name is INGREZZA®) for the treatment of chorea in HD. Initial results from combing KINECT-HD with current KINECT-HD2 patients recently supported the ability of the U.S. Food and Drug Administration (FDA) to approved INGREZZA® capsules for the treatment of adults with chorea associated with HD.
Phase I/II study of AMT-130 in HD
Who runs this study? uniQure
What’s its current status? Active and recruiting.
Where is it running? United States and Poland.
Who’s potentially eligible? Adults between the ages of 25 and 65 with early manifest HD.
What are they investigating? In this trial, uniQure are investigating low and high doses of their drug AMT-130 – a one-time gene therapy candidate administered by neurosurgical procedure, designed to silence the huntingtin gene. This 5-year trial will evaluate the safety and potential impact of AMT-130 on disease progression. Enrolment has been completed for the first two patient cohorts in the US. A third patient cohort is planned for the US to explore an improved administration procedure. An open-label arm of the study remains open to recruitment in Europe.
Phase I/II study of BV-101 in HD
Who runs this study? AskBio
What’s its current status? Not yet recruiting.
Where is it running? Expected to begin in France.
Who’s potentially eligible? Not yet known.
What are they investigating? The BV-101 clinical trial will assess the safety, tolerability, and preliminary efficacy of BV-101 in adults with early-stage HD. The trial will include 12-18 participants and is expected to begin in Paris in Q4, 2022. BV-101 is a gene therapy treatment, aiming to restore cholesterol metabolism, reduce mutant huntingtin and improve neuronal function.
Phase II Study of ANX005 in HD
Who runs this study? Annexon
What’s its current status? Completed.
Where is it running? United States.
Who’s potentially eligible? Adults aged 18 and over with, or at risk for, manifest HD.
What are they investigating? This is an open-label trial of the intravenous drug ANX005. ANX005 is an antibody designed to block an initiator of the immune response, which is known to contribute towards the neurodegeneration seen in HD. Results from this trial showed ANX005 was successful in targeting the complement system. In a subgroup of participants, it appeared the progression of symptoms was slowed, suggesting a sub-population of people with HD might benefit from treatment with ANX005. Annexon will likely conduct a larger trial to confirm these results.
Phase I Study of SKY-0515 Trial
Who runs this study? Skyhawk Therapeutics
What’s its current status? Completed Parts A and B; active and recruiting for Part C
Where is it running? Australia
Who’s potentially eligible? People aged 25 to 65 with early-stage HD (Stage 1, 2, or mild Stage 3)
What are they investigating? This study evaluated the safety, tolerability, and pharmacokinetics of SKY-0515 in healthy volunteers and individuals with early-stage HD. The trial is separated into three parts. Parts A and B evaluated SKY-0515 in healthy volunteers, and Part C will evaluate SKY-0515 in individuals with early-stage HD. The study has successfully demonstrated a reduction of mutant huntingtin in healthy volunteers using participants from Parts A and B. In addition, SKY-0515 was generally well tolerated at all doses tested in healthy volunteers. Given these findings, the company will continue studying dosing with Part C participants with early-stage HD and anticipates initiation of a Phase 2 study early next year.
Phase II Study of SOM3355 in Huntington's Disease Chorea
Who runs this study? SOM Innovation Biotech SA
What’s its current status? Active and recruiting.
Where is it running? Spain (more countries expected to be added soon).
Who’s potentially eligible? Individuals over the age of 21 diagnosed with Huntington's Disease and suffering with choreic movements.
What are they investigating? This trial is assessing the efficacy and safety of SOM3355 in patients suffering from Huntington's Disease with choreic movements. SOM3355 is an oral antihypertensive drug which has been used for years, and is now being tested to treat chorea in HD.
Generation HD2
Who runs this study? Roche
What’s its current status? Active and Recruiting
Where is it running? Opened in North America, Europe, South America and Oceania.
Who’s potentially eligible? People aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD.
What are they investigating? This study evaluates the safety, biomarkers and efficacy trends of different dose levels of investigational drug tominersen in people aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD.
PIVOT-HD
Who runs this study? PTC Therapeutics
What’s its current status? Active and recruiting.
Where is it running? Germany, France, Netherland, & UK.
Who’s eligible? Adults ages 25 and over with a CAG repeat between 42 and 50, with no functional or movement symptoms of HD.
What are they investigating? PTC are investigating their drug PTC518, taken orally, which has been designed to reduce the production of the mutated huntingtin protein. PTC518 has been tested in a Phase I trial in healthy volunteers. PIVOT-HD is a Phase II trial aiming to confirm dose-dependent lowering of huntingtin protein and gain insight to biomarker data that could provide meaningful evidence of treatment effect.
PROOF-HD
Who runs this study? Prilenia
What’s its current status? Completed; Analyzing Data
Where was it running? Austria, Canada, Czech Republic, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States.
Who was potentially eligible? Adults aged 25 and over with early manifest HD.
What are they investigating? PROOF-HD is a Phase III trial to evaluate the efficacy and safety of the drug pridopidine on functional capacity in adults with early manifest HD. Pridopidine is a small molecule drug thought to target a protein called the sigma-1 receptor. Taken orally, it is hoped pridopidine will have beneficial effects on neuronal cells. Preliminary analysis showed the study did not find a significant effect on change in functional capacity when looking across all study participants (the primary endpoint). However, there was evidence of a potential benefit in the subset of patients that were not taking neuroleptics (e.g., antipsychotics) or chorea medications. Additionally, there were other potential benefits shown on secondary endpoints capturing motor scores. Pridopidine was well-tolerated with no serious treatment-related adverse events.
Read Community Letter About Initial Analysis
SELECT-HD
Who runs this study? Wave Life Sciences
What’s its current status? Active and recruiting.
Where is it running? Australia, Canada, Denmark, France, Germany, Italy, Poland, Spain, United Kingdom
Who’s potentially eligible? Adults aged 25 to 60 years with HD.
What are they investigating? This Phase Ib/IIa trial is investigating the safety and tolerability of WVE-003 in people with HD. WVE-003 is a drug designed to lower the toxic form of the huntingtin protein, delivered intrathecally (into the CSF through the spine). WVE-003 targets a specific genetic signature, which not every person with HD has, so if successful this drug would only be able to treat a subset of people with HD.
SURVEYOR
Who runs this study? Sage Therapeutics
What’s its current status? Active and recruiting.
Where is it running? United States
Who’s potentially eligible? Adults aged 25 to 65 with pre or early manifest HD and healthy controls.
What are they investigating? This phase II trial will investigate baseline cognitive performance difference between participants with early HD and healthy participants and the effect of SAGE-718 on cognitive performance and functioning in participants with HD.
VIBRANT-HD
Who runs this study? Novartis
What’s its current status? Novartis recently announced they have stopped the development of Branaplam in Huntington's disease.