There is a continued investment into research on Huntington’s Disease, whether looking at the treatment of symptoms or disease modification. It’s important for the HD community to have access to this information in a clear and conversational manner to better understand what is being studied and how they may become involved if interested. This page summarizes some of the current major clinical trials underway that pertain to the young people that HDYO serves.

We have divided the different clinical trials into two sections:

1. Disease Modiying Trials

2. Symptom Improvement Trials

What is an Clinical Trial?

Also referred to as an interventional study, clinical trials are research studies that involve human participants to evaluate the safety and effectiveness of new treatments, such as drugs, medical devices, or behavioral interventions. Other studies like Enroll-HD, surveys and registries are considered Observational Studies. Check out the Observational Studies page for more on current studies in this domain.

To review other helpful research definitions, explore the HDYO Research Terminology.

Where to Find Trials Use the HD Trial Finder sites below which can provide clinical trial matching services directly. They provide a useful 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 currently 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 and can also be used to find trials. However, this site can be challenging for the community to navigate if they don't have a scientific background. We suggest starting with the different HD Trial Finder sites above first or reviewing the resources below.

Disease Modifying Trials

These studies are trying to modify how Huntington’s Disease progresses in individuals carrying the HD gene. To learn more about these approaches, visit the Current Research Focused on HD section of the HDYO Research Terminology.

Falcon-HD

• 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 & New Zealand
• 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.

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Generation HD2

• Who runs this study? Roche
• What’s its current status? Active
• 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.

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HD-GENETRX2

• Who runs this study? uniQure
• What’s its current status? Active and recruiting.
• Where is it running? United States, United Kingdom 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 is evaluating the safety and potential impact of AMT-130 on disease progression. Interim results from the first two years showed a statistically significant slowing in disease progression among patients receiving the high dose of AMT-130 when compared to an external control group. AMT-130 continues to remain generally well-tolerated, with a manageable safety profile at both doses.

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PIVOT-HD

• Who runs this study? PTC Therapeutics/Novartis
• What’s its current status? Active and recruiting. Completed and analyzing data
• Where is it running? Germany, France, Netherlands, & 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. Interim data from patients who completed 12 months of treatment showed a dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF) as well as favorable effects on several HD-related functioning outcomes. Additionally, PTC519 shows continued safety and tolerability at the 12-month timepoint. Based on these findings, PTC will begin work on the design of the Phase 3 efficacy trial of PTC518.

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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.

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Phase I Study of SPK-1001

• Who runs this study? Roche and Huntington Study Group (HSG)
• What’s its current status? Active and recruiting.
• Where is it running? United States
• Who’s potentially eligible? Adults ages 25-65 who have genetically confirmed HD and mild clinical symptoms.
• What are they investigating? This trial is assessing the safety, tolerability and preliminary efficacy of SPK-10001 in patients suffering from Huntington's Disease. SPK-10001 is a gene therapy that delivers genetic instructions into the brain cells to reduce mutant huntingtin that causes brain atrophy and clinical symptoms affecting motor function, cognition, and behavior for individuals with HD. In this study, the researchers will slowly increase the dose for each small group of participants to find the best dose of a study treatment for participants to receive.

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SELECT-HD

Who runs this study? Wave Life Sciences What’s its current status? Completed and Analyzing Data Where did it run? Australia, Canada, Denmark, France, Germany, Italy, Poland, Spain, United Kingdom Who’s potentially eligible? Adults aged 25 to 60 years with HD. What were they investigating? This Phase Ib/IIa trial investigated 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 (through the spine). The study demonstrated that WVE-003 selectively lowered the toxic, mutant huntingtin protein while preserving healthy, wild-type huntingtin protein for individuals with Huntington’s disease. It also showed that WVE-003 was generally safe and well-tolerated. Based on these positive findings, Wave is planning activities for a global Phase 2/3 study of WVE-003.

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VO659

• Who runs this study? Vico Therapeutics
• What’s its current status? Active and recruiting
• Where is it running? Europe
• Who’s eligible? Adults ages 25 to 60 early manifest HD
• What are they investigating? This Phase 1/2a clinical trial is designed to assess the safety and tolerability of four doses of VO659 administered intrathecally (through the spine) every four weeks in participants with early manifest HD. VO659 has a unique allele-preferential mechanism of action that targets the underlying CAG repeat expansion that causes HD. Interim data suggest that VO659 may be able to reduce levels of the toxic HD protein in the small group of participants tested so far. Still, interim data presented also showed that 4 (out of 6) people who received at least three doses of 40 mg experienced radiculitis, a painful condition related to nerve damage. Vico plans to mitigate this issue by lowering the amount of drug given to people in the trial.

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Symptom Improvement Trials

These studies are targeted at suppressing symptoms such as those caused by chorea, cognitive decline and behavioral changes. They are not modifying disease course, but aim to improve daily life instead in regards to symptom severity.

KINECT-HD2

• Who runs this study? Neurocrine Biosciences and the Huntington Study Group
• What’s its current status? Active.
• Where is it running? Canada and United States.
• Who’s potentially eligible? Adults aged 18 to 75 with a diagnosis of motor manifest HD.
• Which symptom(s) is being targeted? Chorea
• 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 combinig KINECT-HD with current KINECT-HD2 participants recently supported the ability of the U.S. Food and Drug Administration (FDA) to approve INGREZZA® capsules for the treatment of adults with chorea associated with HD. More recently, Neurocrine has introduced an oral granules formulation of INGREZZA (called INGREZZA® SPRINKLE) that provides an alternative administration option for those who have difficulty swallowing or prefer not to swallow whole capsules.

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PROOF-HD

• Who runs this study? Prilenia
• What’s its current status? Completed; Analyzing Data
• Where did it run? 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.
• Which symptom(s) is being targeted? Chorea, Cognition and Behavioral
• 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.

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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 choreatic movements. Which symptom(s) is being targeted? Chorea What are they investigating? This trial is assessing the efficacy and safety of SOM3355 in patients suffering from Huntington's Disease with choreatic movements. SOM3355 is an oral antihypertensive drug which has been used for years, and is now being tested to treat chorea in HD.

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