Clinical Trials

Designed to test the safety and effectiveness of treatments, clinical research depends on the participation of volunteers with HD. Participants are typically divided into at least two groups, usually randomly: one that receives the treatment and one that does not.

Clinical trials are time-limited, so participants can join, or enroll, in the studies only when they are recruiting. Historically, clinical trials for Huntington’s disease have only included people who are gene positive and symptomatic. Due to findings from some recent research, those who are gene positive and not yet fully symptomatic, or are early in the disease, are now also being included in HD clinical trials.

Stages of Clinical Trials

Learn more about clinical research from the Huntington’s Study Group. (external link)

https://neurosciences.ucsd.edu/centers-programs/huntingtons-disease/
research/clinical-observational-trials.html

Phase I

The goals of this first step in testing a new treatment or drug in humans are to make sure the treatment is safe, and to figure out the right dose to use in future studies. Patient volunteers are followed primarily for side effects, not for how the drug affects their disease. The first few volunteer subjects receive low doses of the trial drug to see how the drug is tolerated and to learn how it acts in the body. The next group of volunteer subjects receives larger amounts. A small number of “healthy” volunteers are also included.

Phase II

Involves a drug whose dose and side effects are well known. Many more volunteer subjects are tested to define side effects, learn how it is used in the body, and learn how it helps the condition under study.

Phase III

Compares the new drug against a commonly used drug or placebo. Some volunteer subjects will be given the new drug and some the commonly used drug. The trial is designed to find where the new drug fits in managing a particular condition.