HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease
Home-based TDCS (Transcranial Direct Current Stimulation) for Cognitive and Behavioral Symptoms in Huntington's Disease
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A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
What is Huntington's disease?
Huntington's disease is an inherited brain disorder caused by an expanded stretch of repeated DNA, called CAG repeats, in the huntingtin gene. Most people have around 20 repeats; people who develop the disease usually have about 40 or more. It causes involuntary movements called chorea, gradual loss of thinking and planning ability, and mood and personality changes, usually beginning between ages 30 and 50 and progressing over 10 to 20 years. Each child of a parent with the gene has a 50 percent chance of inheriting it.
About 41,000 Americans have symptoms and more than 200,000 are at risk. A blood test can identify gene carriers before symptoms appear, and testing is done with genetic counseling because the result affects the whole family. Current medicines treat chorea, mood, and sleep, but none slows the disease, which is the goal of today's trials. Movement and memory changes overlap with Parkinson's disease and other dementias.
Types of Huntington's disease clinical trials
Disease modifying drug trials. Phase 3 studies of oral medicines meant to slow progression, measured over years by movement, thinking, and daily function.
Gene therapy and huntingtin lowering trials. Treatments delivered into the brain or spinal fluid that reduce the harmful protein; see gene therapy clinical trials.
Cell therapy trials. Early safety studies of neural stem cells.
Symptom trials. New treatments for chorea, irritability, sleep, and swallowing.
Biomarker and observational studies. Spinal fluid, imaging, and digital measures that enroll gene carriers without symptoms, people with symptoms, and family members, building the tools future trials depend on.
Care and caregiver studies. Programs for families, who carry much of the disease's weight.
New to research? Read how informed consent works, including when a person's ability to decide changes over time.
Who can participate in Huntington's disease clinical trials?
Treatment trials usually enroll adults in a set age range, often 21 to 70, with a confirmed CAG expansion and early to moderate symptoms measured on a standard rating scale; some enroll people at the very first signs. Gene therapy trials are small, involve brain surgery or spinal injections, and have strict rules. Observational and biomarker studies are the widest door: many enroll gene carriers with no symptoms, people at risk who have not been tested, and family members. Most studies ask that a study partner, often a family member, comes to visits. Eligibility always varies by study.
Stages of Huntington's disease, and why they matter for trials
Premanifest. Gene carriers without symptoms, sometimes for decades. Observational studies and, increasingly, prevention trials enroll here.
Early. Subtle movement or thinking changes through mild symptoms. The main target of disease modifying trials, because there is most to protect.
Moderate. Clear chorea and cognitive change, with help needed for some tasks. Some drug and symptom trials enroll.
Advanced. Full care needed. Mostly care and symptom studies.
Is there a cure for Huntington's disease?
Not yet. Approved medicines treat chorea and mood symptoms but do not slow the disease. Treatments that lower the harmful huntingtin protein, including a gene therapy that reported slowed progression in an early trial in 2025, are the closest thing to a cure in development.
Do Huntington's disease trial participants get paid?
Some studies offer compensation for time and travel, and study related care is typically provided at no cost to participants. Compensation varies by trial and is always described during the informed consent process before you agree to anything.
Curious whether clinical trials pay participants? Here's how compensation actually works.
How to enroll in a Huntington's disease clinical trial
Identify your trial. Use the filters; titles say "early manifest," "premanifest," or name a symptom.
Select your preferred location. Enter your city or state. Phase 3 trials run at dozens of specialist centers; gene therapy at a few.
Explore study details. Click "Learn More" for genetic test requirements, stage, and whether a study partner must attend.
Complete the health profile. Click "Get started" to begin the 5-step application. Have ready your genetic test result if you have one, when symptoms began if they have, and your current medicines; a family member can complete it with you.
Submit the application. A clinical trial coordinator reviews it and contacts you. Nothing is decided until informed consent, and participation is voluntary at every step.