Clinical Trials

A clinical trial is a study of a treatment in volunteer patients. A clinical trial will determine the right dose and frequency of the treatment. It will test whether the treatment is safe and effective, and will determine whether any side effects are tolerable or easily managed.

Developing a new treatment is a long process that can take between five and seven years. The Food and Drug Administration (FDA) oversees every step of the process.

Clinical trials typically have three phases:

For clinical trials on treatments for rare diseases, the FDA will often allow the first two phases to be combined because there are so few patients.

The best way to find out if you or your family member is eligible for a clinical trial is to speak with the study investigator to discuss the study objectives, expectations for participation, and potential risks and benefits. Be sure to sign an informed consent form before undergoing further screening, assessments or administration of treatments.

For most clinical trials involving rare diseases, there is no cost to participate. The treatment, hospitalization, and assessments are free of charge. Usually, the clinical trial will pay for the patient’s travel, hotel, and meals—and often will cover these charges for a companion or guardian.

Visit www.clinicaltrials.gov, which lists most clinical trials in the United States with eligibility criteria and participation requirements.

You can also find clinical trials in the United States that are seeking patients with Tay-Sachs, Canavan, GM1 gangliosidosis, or Sandhoff diseases by selecting the tabs in the table below.

We will update this listing when new clinical trials launch.

The webinars below include more information on clinical trials and the role of the FDA in the process.

If you’re thinking about taking part in a clinical trial and want more information, you are always welcome to email Diana Jussila (diana@ntsad.org), Director of Family Services, or Becky Benson (becky@ntsad.org), Family Services Manager.