Clinical Trials and Research

Some of the world’s top scientists are hard at work to develop treatments for Huntington’s disease – and real progress is being made. Dozens of possible treatments are being investigated and hope is building in the HD community.

Get Involved

Successful treatments will arrive more quickly if more people help out.  Here’s how you can get involved:

UW Clinical Trials

Observational Studies


Enroll-HD is the world’s largest observational study for Huntington’s disease families—currently more than 20,000 people are taking part in Europe, North America, Australasia, and Latin America. By monitoring how the disease happens in people and how it changes over time, researchers can use the huge data and biosample collection to learn more and come up with new ways to effectively treat HD.

Enroll-HD is open to anyone who has HD or is at risk. The study requires an in-person clinic visit once per year that may involve cognitive and mood assessments, blood draws, questionnaires, motor examinations, and the collection of demographics and health information. This study will help guide the development of treatments and therapeutics for HD patients in the coming years. For more information, please follow the link to the enroll HD website.

Enroll HD:

Study Contact: Amy Good


Clinical Trials


UniQure is a clinical trial that aims to explore the safety, tolerability, and efficacy regarding the Striatal administration of the therapy AMT-130 in patients with Early Manifest Huntington’s Disease. AMT-130 is an experimental gene lowering therapy that may lower the Huntington protein level resulting in the slowed progression of an individuals’ HD. The administration of AMT-130 requires a neurosurgical procedure.

This study will soon enroll the final Cohort late in 2023 in an attempt to shorten surgical times for this procedure. Participation in the study will last up to 60 months (about 5 years) and includes clinic visits, assessments of physical and neurological health, lumbar punctures, a neurosurgical procedure, brain scans, and biological sample collection

Additional Information: Study Record |

Study Contact: Debra Del Castillo



PROOF-HD is a research study that is investigating the use of the drug Pridopidine as a possible treatment for Huntington Disease (HD). Researchers aim to evaluate the long-term safety and tolerability of Pridopidine, as well as its impacts on everyday functional capacity, movement, and behavior in participants with early-stage Huntington Disease. This study completed enrollment in the summer of 2018. Participants are now in the Open-Label (OLE) period to assess long-term effects of Pridopidine. During the OLE period no participants will be given the Placebo or inactive form of the drug. The main research activities include questionnaires, cognitive and mood examinations, and health history.

Additional Information: Study Record |

Study Contact: Debra Del Castillo


Other Studies

HD Trial Finder

Use HDSA’s HD Trial Finder to locate Huntington’s disease clinical trials nationwide.


Researchers, clinicians and other HD healthcare professionals frequently need to solicit input from the HD patients, families and caregivers through anonymous surveys. The results of these surveys are used to help guide future HD care and research strategies. Learn more about current surveys that could benefit from your feedback.

Get Up to Speed on Promising Research

HD Buzz

HD Buzz – “Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.”

Dr. Ed Wild and Dr. Jeff Carroll (co-founders of HDBuzz) gave this fantastic Avengers-themed presentation reviewing the status of HD research at the 2019 HDSA Annual Convention. It’s an easy to follow talk, packed with plenty of superhero references and jokes.


Stay up to date on HD Research by following HDSA Research communications – including a weekly blog, webinars, annual research reports and more!

Huntington Study Group

The Huntington Study Group (HSG) is a world leader in facilitating high-quality clinical research trials and studies in Huntington disease (HD).