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Huntington’s disease Treatment Development

 Huntington’s disease research is constantly adding more potential drug treatments into the pipeline. The clinical phases of research take time and must go through specific phases before it can be prescribed by a physician in an HD clinic.

Phases of Clinical Trial Development

Click on each of the phase boxes to see active HD studies for each phase.

HD has more drug candidates in the pipeline than ever before. Click below to view additional studies happening around the world. Once you are on that site, you can filter your study search by geography, sex, age, phase, and other attributes that are most applicable to you. Some studies list their phase as “Not Applicable”. This is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions that do not involve drugs, or observational studies.

Types of HD Treatments

There are over 50 companies studying therapies for HD. Treatments can be thought of in several ways:

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Treatments differ in what they do. Some can change the course of a disease while others only treat symptoms. Some change chemical signals in the body (neurotransmitters), or the production or action of the body’s proteins such as the protein responsible for HD. There are also drugs that try to prevent brain or nerve degeneration.

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Stage of product development

Where is the drug in its research lifespan?
A drug must pass through 5 phases with the FDA: 1) discovery and development, 2) preclinical research, 3) clinical research, 4) FDA review, and 5) safety monitoring.

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Route of administration

This describes how a drug is given to patients. For example, it may be by mouth (oral) or some other method (parenteral) such as into a vein (intravenous), into the brain (intracranial), or around the spinal cord (intrathecal).


Molecule Type

Small molecule – the most common type of drugs, these use components synthesized from chemicals in a lab to treat or prevent diseases

Large molecule (biologics or biosimilars) – these are derived from a natural source including humans, animals or microorganisms and include monoclonal antibodies, gene therapies, and interleukins.

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Other studies

Though we often focus on drugs, there are also studies that examine non-drug interventions such as physical therapy, music, diet, exercise, and behavioral therapy. Often, these studies have the goal of improving someone’s quality of life and reducing symptoms that may be bothersome.

Click here to learn about current HSG trials.

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