About Clinical Trials
What is a clinical trial?
A clinical trial is a way for people in the medical community to find out if a treatment is safe and effective to treat a medical problem like cancer.
Clinical trials involve the treating doctors and medical team at a specific clinic participating in the clinical trial, plus the study team from the company that makes the treatment, and health authorities like the FDA.
After the clinical trial is completed, and if the treatment works and is safe, the information from the clinical trial is given to the FDA. FDA then carefully reviews the information and makes a decision whether or not to approve the treatment for the disease that was studied in the clinical trial.
What is a basket trial?
Traditional clinical trials focus on a specific type of cancer, such as breast cancer or lung cancer. By contrast, a basket trial tests how well a new drug works in patients who have different types of cancer that all have the same alteration or biomarker. In basket trials, patients all receive the same treatment that targets the specific mutation or biomarker found in their cancer. Basket trials can also be useful for studying rare cancers and cancers with rare genetic changes.
What is a biomarker?
A biomarker is a biological molecule found in blood, other body fluids, or tissues that is a sign of a condition or disease. The biomarkers being used in the PRECISION 1 trial are certain alterations in the TSC1 and TSC2 genes. Visit the National Cancer Institute for more information on biomarker testing.
What is a just-in-time trial?
In traditional trials, a set number of trial sites are activated and then begin to look for eligible patients. In just-in-time trials like PRECISION 1, potential sites pre-identify patients, and can be activated quickly as soon as there is a patient need. Often, just-in-time trials use networks of oncology centers that have access to many types of cancer patients.