Understanding your Clinical Trial options

Clinical trials are research studies that involve people, and test new medications or medical equipment to find out if they are safe and effective for patients. Nearly all of the standard cancer treatments used today are based on the results of clinical trials. Patients who participate in clinical trials help improve cancer treatments for future patients. The information gained from many clinical trials is used by the U.S. Federal Drug Administration (FDA) to approve new drugs and devices for patients.

In the United States, all medical devices and drugs must be approved by the FDA for use by the general public. All patients who participate in clinical trials are volunteers and may choose to stop their participation in the trial at any time. Clinical trials are generally conducted in specific steps, called phase I, II, III, and IV.

Phase I trials learn whether a product is safe or not. Usually, the goal is to find the highest dose of the drug that is safe and the best way to give it, without creating side effects that would be harmful to patients. In phase I trials, also known as dose-finding or dose-escalation trials, patients may receive very low doses of the drug being studied. The dose is gradually increased in several groups of patients until patients begin to have side effects. Once the maximum tolerated dose (MTD) of the drug is identified, the study is stopped. The dose found in the phase I study is then used in the next phase II trial. In general, patients are offered participation in Phase I trials only if they have already received treatments that are known to be effective for their particular cancer.

Phase II trials find out whether a new treatment works against the cancer that is being studied. Generally, phase II trials study drugs that have not yet been approved by the FDA (investigational drug) or are approved for use in a different type of cancer. All patients receive the drug being tested.

Phase III trials compare the new treatment to the current “standard” treatment to see if it gives a better result for patients. In comparative trials, each patient is randomly assigned to one of the treatment plans being studied (randomized clinical trial). Often in phase III trials, the doctor and the patient do not know which treatment the patient is receiving (‘blinded’ study), and the treatment plan may include a placebo ("dummy" drug that has no effect) .The purpose of randomization, blinding and placebo is to remove any bias from the trial, so that comparisons can be made fairly.

The goal of a phase IV trial is to gather more information about a new treatment after it has been approved by the FDA.

What does participating in a clinical trial involve?

When a patient is offered a clinical trial as a treatment option, they will go through a process called ‘informed consent’. Informed consent includes reading a short document that describes the study, and a discussion with the doctor to help patients learn about a clinical trial and help them decide whether to participate. This information includes what is involved, the purpose of the study, the tests and the other procedures used in the study, and the possible risks and benefits. Any clinical trial offered to a patient, has gone through a rigorous formal review and approval process to make certain the research is valuable, ethical, and that patients' safety and privacy are protected.

Possible benefits of participating in a clinical trial include:

Access to promising new drugs or approaches that may not be available outside the clinical trial.
Better treatment results compared to "standard" treatment.
Results from the study may help others in the future.
Thorough medical attention from the doctor and the research team.

Possible risks of participating in a clinical trial include:

New drugs or procedures under study may not be better than the standard care.
New treatments may have side effects or risks that doctors do not expect.
In randomized trials, patients are not able to choose the therapy that they receive.
More visits to the doctor or tests may be required in a clinical trial.

One of the best sites for researching clinical trial options is www.clinicaltrials.gov

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Understanding your Clinical Trial options