Clinical Trials

A clinical trial is a study that compares a specific treatment that is currently recognized as the best available (standard of care) with a treatment that the study's researchers believe is possibly more effective or even safer. If a clinical trial proves that a new treatment is more effective than current therapies, then it may become the new standard of care. It is through clinical trials that we make progress in the battle against cancer and that patients receive 'cutting edge' therapy.

Below are factors to consider before enrolling in a clinical trial:

Pros

Cons

Phases

Phase I These small trials are the first step in testing new treatments in patients. The goal is to determine how the treatment can be given, for example the best route of administration and appropriate dose of a new drug, and to monitor how the patient responds. Because less is known about the possible risks and benefits in Phase I trials, these studies usually include only a limited number of patients who would not be helped by other known treatments.

Phase II In phase II, the researchers examine the efficacy of the treatment, (e.g. tumor shrinkage), and look for side effects. Although larger than Phase I trials, limited numbers of patients enter phase II trials because the efficacy and the side effects of the new treatments are still largely unknown.

Phase III trials compare the results of people taking the new treatment with results of people taking standard treatment. Questions are asked such as: Which group has better survival rates? Which group has fewer side effects? Patients are randomly divided into each treatment group, one receiving the new treatment, and the other receiving the current standard of care treatment. Sometimes patients do not know which group they are in (a blinded trial). In most cases, studies move into Phase III testing only after a treatment shows promise in Phases I and II. Phase III trials may include many hundreds of people around the country.

Safety

High profile concerns in gene therapy trials has focused public attention on the safety of clinical trials. Cancer clinical trials are tightly regulated and closely monitored by the Federal Government (http://ohsr.od.nih.gov/) to ensure that each trial is performed in the safest manner possible. Below are the safeguards and patients' rights that protect those enrolled in clinical trials.

Institutional Review Board (IRB). All clinical trials must follow a detailed plan, or 'protocol', that is written by the researchers and must be approved by the IRB at each institution before implementation of the clinical trial. This board, which includes consumers, clergy, and health professionals, reviews the protocol to try to be sure that the research will not expose patients to extreme or unethical risks.

Informed Consent. A patient must receive all the facts about a study before deciding whether to take part. This includes details about the treatments and tests you may receive and the possible benefits and risks they may have. The doctor or nurse will give you an informed consent form that goes over key facts. If you agree to take part in the study, you will be asked to sign this informed consent form. During the course of the trial, you will be told of any new findings regarding your clinical trial, such as new risks. You may be asked to sign a new consent form if you want to stay in the study. Signing a consent form does not mean you must stay in the study. In fact, you can leave at any time. If you choose to leave the study, you will have the chance to discuss other treatments and care with your own doctor or a doctor from the study.

Leave trial at any time. You have the right to leave a study at any time. If you do enter a study, doctors and nurses will follow your response to treatment carefully throughout the research. If researchers learn that a treatment harms you, you will be taken off the study right away. You may then receive other treatment from your own doctor.

Eligibility criteria. To enter a clinical trial, a patient must meet the conditions, or eligibility criteria, described in the protocol. These criteria differ between studies, but often include type of cancer, prior treatment, extent of disease. This ensures that the trial will yield useful information and that only patients with appropriate diseases enter the trial.

Taking part in a treatment study is up to you. It may be only one of your treatment choices. Talk with your doctor and together decide if a clinical trial is the best choice for you. If you are interested in finding clinical trials, the web sites below may help.

Finding a Trial

Johns Hopkins provides these resources as a tool. We have no control over the non-Hopkins sites and do not vouch for their accuracy.