Clinical Trials
Clinical trials explore new ways to prevent, find, and treat cancer. Most brain tumor clinical trials are treatment studies that are designed to evaluate whether a new drug or treatment is effective at treating specific types of cancer and safe to give to patients.
What Are Clinical Trials?
Clinical trials are research studies developed to find out how well new medical approaches work to safely and effectively treat people with specific diseases.
When doctors find a drug or treatment that they think might improve the standard approach to treating a disease, such as cancer, they organize a clinical trial to learn what happens when they give the drug or treatment to a group of patients who have a similar disease.
Clinical trials help doctors learn which drugs and treatments are safe to give to patients and which do the best job treating cancer. Patients and their families get to choose whether or not they want to take part in clinical trials. Taking part in a clinical trial is always voluntary and it is an important decision. Be sure to talk through all of your questions with your doctor and family members when you are thinking about whether to enroll your child in a clinical trial.
The Phases of Clinical Trials
Phase 1 Clinical Trials
A Phase 1 clinical trial is the first stage of testing a new drug or treatment in humans. Before a phase 1 clinical trial for a drug can start, the drug has to go through preclinical studies. These are studies that test the drug in cellular (in vitro) and animal (in vivo) models. This helps the drug have the best chance of safety and success for patients. Phase 1 clinical trials can start after their preclinical results are approved for patient safety and treatment success. These trials mostly focus on evaluating safety, tolerability, dosage range and side effects. Even though researchers hope that the drug helps treat cancer, that is not the main goal in phase 1.
First, they need to find out the highest dose of the drug they can give without causing side effects that are too serious. They give the drug to a group of patients and watch closely for side effects. Patients who take part in a phase 1 trial have often tried other drugs and treatments that have not effectively treated their cancer.
Phase 2 Clinical Trials
Once doctors learn the dose of a drug or treatment that is safe to give to children, they move on to a phase 2 trial.
In a phase 2 trial, the drug or treatment is given to a small group of patients, and the goal is to find out if the drug works to slow or stop a patient’s cancer. For a patient with a brain tumor, they give the drug and then watch to see if the tumor stops growing or shrinks.
They also continue to assess safety, determine the best dosing and further study potential side effects.
Phase 3 Clinical Trials
In a phase 3 trial, doctors give a drug or treatment that seems to work for a larger group of patients.
The goal of a phase 3 trial is to find out if the drug they are testing works better than other drugs for the same disease. They give the drug to patients and compare it to other kinds of treatments to find out which is best at treating cancer. These comparisons look at how effective the treatment is at killing cancers cells while having the minimal amount of negative side effects.
Working this way takes time, but it helps doctors learn which treatments are best for treating brain tumors in children.
Finding a Clinical Trial for Your Child
When doctors want to learn which drug or treatment works best for children, it isn’t enough to try it on one child. Doctors need to work with large numbers of patients.
To put together groups that are big enough to show which treatments work best, doctors from many different hospitals work together in clinical trials. They give the same treatment to a group of patients who have tumors that are alike. Working together, doctors can find answers faster than if they work alone.
Understanding whether or not your child qualifies for a clinical trial can be confusing. The Pediatric Brain Tumor Foundation works closely with other organizations to support families wanting to learn more. Complete this brief form or email us at [email protected] to speak with our team about clinical trials.
