Georgetown University Opens Advanced Study for Huntington’s Disease

Media Contact
Karen Teber, km463@georgetown.edu

WASHINGTON (June 24, 2026) — Georgetown University Medical Center has launched a new clinical research opportunity for people diagnosed with Huntington’s disease (HD) who are experiencing the early symptoms of the disease.

The INVEST-HD international study is a Phase 3 research study evaluating an investigational oral medication called votoplam. The goal of the study is to see if this treatment can help slow the progression of HD symptoms. Earlier studies suggest votoplam targets the body’s genetic instructions to stop the production of the toxic huntingtin protein before it can damage nerve cells.

Georgetown, a Huntington’s Disease Society of America Center of Excellence, is the first site in the world to activate, screen and enroll participants in the INVEST-HD trial. This milestone reflects Georgetown’s commitment to providing patients with the earliest possible access to innovative research.

“Georgetown continues to offer the latest treatment studies to our HD community, and we’re especially excited about the potential of this study,” says Karen Anderson, MD, the lead investigator for the study and director of Georgetown’s Huntington Disease Care, Education, and Research Center. “This trial is designed for people diagnosed with Huntington’s disease who have no symptoms or those who have very early symptoms. Participants will receive personalized care through expert medical monitoring and comprehensive health assessments conducted by our specialized team.”

Huntington’s disease is an inherited condition that causes the progressive breakdown of nerve cells in the brain and leads to significant cognitive and motor function impairment. It is a progressive illness, meaning symptoms worsen over time, and there is no cure.

People who are between the ages of 21 and 70 with a previous genetic diagnosis for Huntington’s disease may be eligible for the INVEST-HD trial. The study lasts 24 to 36 months.

The study is designed so that participants are randomly assigned to receive either the investigational drug, votoplam, or a placebo, which looks like the study drug but contains no active medicine. Participants have a 60% chance (3 in 5) of being assigned to receive votoplam. For those who complete the double-blind treatment period, there is the potential to join an “Open-Label Extension” study, where all eligible participants will receive the active medication.

Participation comes at no financial cost, as the study drug and all research-related tests and procedures are provided free of charge. Participants and their study companions will be compensated for their time and effort. Additionally, customized travel and expense support may be available.

Every clinical trial has risks. The most common side effects noted in past studies with votoplam were headaches and falls, with others including dizziness or digestive changes. The clinical trial team will discuss all potential risks and potential benefits.