Understanding Clinical Trials
A clinical trial is a way of testing a new type of medical treatment. Enrollment to clinical trials is voluntary and once you enroll in a clinical trial, you can stop participating at any time.
Benefits, Risks, and Alternatives to Joining a Clinical Trial
The main potential benefit to participating in a clinical trial is that you may have access to a new treatment that might be better than treatment available outside of a clinical trial. However, it is unknown at the start of the trial whether the new treatment will end up being better or not. Another benefit is that you will be helping the medical community learn more about potential new treatments for childhood cancer.
There are risks and disadvantages to taking part in a clinical trial. First, the new treatment may have serious side effects. Second, the new treatment may end up being less effective at treating your cancer compared to standard treatment. Third, there may be more tests and clinic visits needed as part of the trial than would be needed if you were receiving standard treatment. Finally, clinical trials need to monitored very closely to make sure they are being conducted properly and ethically. Therefore, people outside of your medical team may need to review your medical record resulting in a potential risk to your privacy and confidentiality.
The main alternative to participating in a clinical trial is to receive standard care prescribed by your doctor.
You will need to weigh the specific benefits, risks, and alternatives of a trial before deciding whether you want to join the trial.
How to Join (or "Enroll" onto) a Clinical Trial
Before you can join a clinical trial, the study doctor needs to explain the potential risks, benefits, and alternatives of participating in the trial. She or he will review this information along with the written permission form describing the trial (consent form). If you have any questions at all, you should ask them at this meeting. If you would like to participate, the study doctor will ask you to sign the permission form allowing you to be treated on the study. Signing the permission form does not yet mean that you have joined the study. Your study doctor needs to complete tests to ensure that you meet all of the requirements to officially join the study. If you meet all of the requirements, then you can enroll on the trial and start the treatment.
Types of Clinical Trials
Clinical trials fall into three main categories: phase 1, phase 2, and phase 3.
Phase 1: The goal of a pediatric phase 1 clinical trial is to learn about the side effects of the treatment in young people and pediatric safe dose of a new drug or new drug combination. These trials usually treat 3-6 patients at a time with the same dose of the drug. If that dose of the drug is found to be safe, then the dose is increased and another 3-6 patients treated at the higher dose. This process is repeated until the highest safe dose is found. Almost all drugs have already been tested in adults before they are tested in younger people.
Since the side effects and safe dose of the drug are not yet known at the start of a phase 1 trial, these trials are typically only used if initial treatment was not effective.
Phase 2: The goal of a phase 2 clinical trial is to learn whether a new drug or new drug combination is effective. These trials usually treat about 10-20 patients with a specific disease to find out how many patients show improvements in their cancer.
Since drugs being tested in phase 2 trials are still unproven, these trials are typically used if initial treatment was not effective.
Phase 3: The goal of a phase 3 clinical trial is usually to compare a new treatment to the current standard treatment. These trials often involve a process called randomization. Randomization means that the study determines whether you will receive the new treatment or the standard treatment. Phase 3 trials usually treat several hundred patients.
Phase 3 trials are typically used in the treatment of patients at the time of initial diagnosis.