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HD Clinical Trials

9 mai 2022

Huntington's Disease Youth Organization

HDYO met sur son site plus d'informations à la dispoisition de jeunes, parents et professionnels.

www.hdyo.org

Clinicaltrials.gov

Clinicaltrials.gov is a website that lists research studies around the world. It can be useful to find Huntington's Disease studies going on in your country.

Visit clinicaltrials.gov

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. 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

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.

Where can I find out more? https://medicine.uiowa.edu/psychiatry/research/change-hd https://clinicaltrials.gov/ct2/show/NCT01860339

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.

Where can I find out more?
https://enroll-hd.org/ https://clinicaltrials.gov/ct2/show/NCT01574053

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.

Where can I find out more? https://hdclarity.net/ https://clinicaltrials.gov/ct2/show/NCT02855476

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.

Where can I find out more? https://www.lumc.nl/org/neurologie/research/hd-junior/

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.

Where can I find out more? https://join-hd.org/

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.

Where can I find out more? https://medicine.uiowa.edu/psychiatry/research/kids-jhd

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.

Where can I find out more? https://neurology.wisc.edu/prevent-hd/
https://clinicaltrials.gov/ct2/show/NCT04818060

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.

Where can I find out more? https://www.triplettx.com/patients/ https://clinicaltrials.gov/ct2/show/NCT04406636

Interventional Research

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.

Where can I find out more? https://perspectivehdprogram.com/
https://clinicaltrials.gov/ct2/show/NCT05107128

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 for the treatment of chorea in HD.

Where can I find out more? https://huntingtonstudygroup.org/current-clinical-trials/kinect-hd2/
https://clinicaltrials.gov/ct2/show/NCT04400331

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. Administration of high dose AMT-130 has been postponed due to recent suspected unexpected severe adverse reactions.

Where can I find out more? http://uniqure.com/patients/phase-1-2-clinical-trial-of-amt-130.php
https://clinicaltrials.gov/ct2/show/NCT04120493 https://clinicaltrials.gov/ct2/show/NCT05243017

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.

Where can I find out more? https://www.askbio.com/brainvectis-a-subsidiary-of-askbio-receives-clearance-to-conduct-phase-i-ii-clinical-trial-in-france-for-its-novel-gene-therapy-for-early-stage-huntingtons-disease/

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.

Where can I find out more? https://ir.annexonbio.com/news-releases/news-release-details/annexon-biosciences-reports-phase-2-clinical-trial-results
https://clinicaltrials.gov/ct2/show/NCT04514367

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.

Where can I find out more? https://sombiotech.com/pipeline/ https://clinicaltrials.gov/ct2/show/NCT05475483?term=SOMCT03&draw=2&rank=1

Phase II Study of tominersen in HD

Who runs this study? Roche

What’s its current status? Not yet active.

Where is it running? Not yet known.

Who’s potentially eligible? Not yet known.

What are they investigating? Following dosing being stopped in GENERATION-HD1 (a phase III trial of tominersen) in 2021, Roche announced in early 2022 that they will continue investigating tominersen in HD through a new phase II trial - GENERATION-HD2. This global study will evaluate the safety, biomarkers and efficacy of tominersen in people aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD.

Where can I find out more? https://hdsa.org/wp-content/uploads/2022/09/Roche-Global-Patient-Community-letter_EHDN-2022.pdf

PIVOT-HD

Who runs this study? PTC Therapeutics

What’s its current status? Active and recruiting.

Where is it running? Germany, United States.

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.

Where can I find out more? https://www.ptcbio.com/our-science/therapeutic-areas/about-huntingtons-disease/
https://clinicaltrials.gov/ct2/show/NCT05358717

PROOF-HD

Who runs this study? Prilenia

What’s its current status? Active, not currently recruiting.

Where is it running? Austria, Canada, Czech Republic, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States.

Who’s 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.

Where can I find out more? https://www.prilenia.com/what-is-huntingtons-disease
https://clinicaltrials.gov/ct2/show/NCT04556656

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.

Where can I find out more? https://clinicaltrials.gov/ct2/show/NCT05032196?term=WVE-003-001&draw=2&rank=1

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.

Where can I find out more? https://perspectivehdprogram.com/
https://clinicaltrials.gov/ct2/show/NCT05358821

VIBRANT-HD

Who runs this study? Novartis

What’s its current status? Active, not recruiting, dosing suspended.

Where is it running? Belgium, Canada, France, Germany, Hungary, Italy, Spain.

Who’s potentially eligible? Adults between the ages of 25 and 75, with early manifest HD.

What are they investigating? This is a Phase II trial from Novartis of their drug branaplam to determine the correct dose required to lower mutant huntingtin protein levels in CSF in adults with early manifest HD. Branaplam is an oral drug designed to reduce mutant huntingtin protein by altering mRNA splicing. Branaplam was initially developed for children with spinal muscular atrophy and has been tested in healthy adult volunteers in a Phase I trial. Dosing has been suspended due to concern of potential peripheral neuropathy in some participants.

Where can I find out more? https://www.novartis.com/news/novartis-receives-fda-fast-track-designation-branaplam-lmi070-treatment-huntingtons-disease
https://clinicaltrials.gov/ct2/show/NCT05111249