What Are Clinical Trials?

Clinical Trials as a Part of Cancer Treatment

Jan 13, 2008

Find out more about what clinical trials are, how they are conducted, how they can affect your cancer treatment, and why they are an important part of cancer research.

What Are Clinical Trials?

Clinical trials are special research studies designed to test promising new medical treatments. The research being conducted may be evaluating new methods for cancer prevention, new diagnostic procedures, or new treatments. Clinical trials also test new ways of using existing treatments for cancer. Clinical trials are often sought out by melanoma patients when the few traditional treatments that are available aren't working for them.

As clinical trials prove new treatments to be safe and effective, these new treatments join the others as standards of care. The treatments for cancer that are now highly respected as standards of cancer care were previously proven effective by similar clinical trials.

What Are the Benefits of Participation?

Having access to new treatments that are not available elsewhere.
New treatment might be much more effective than current treatments available.
Treatment performed by a team of highly trained professionals.
Results of the study may help many others in the future.

What Are the Risks of Participation?

New therapy may be less effective than the current standard of care.
Risks and harmful side effects may be worse than with the current standard of treatment, or even completely unexpected.
Insurer may not cover some of the investigational treatment, or any at all.

Phases of Clinical Trials

Clinical trials involving new cancer therapies progress through a series of steps called phases. Having these separate phases allows researchers to learn more about the safety and effectiveness of the new therapy or drug in a progressively more specific way while also safeguarding the safety of the patients involved in the study.

Phase I Trials

This is the first testing of a new drug or therapy on humans. Usually enrolling only a small number of participants, phase I trials are seeking answers to these types of questions:

What is a safe dosage amount?
Which route of administration is the safest -- by mouth, injected into muscle, or injected into the vein?
How often can this be given -- once per day, twice per day, every 6 hours?
Are there harmful side effects?

Most often the highest dose with acceptable levels of side effects will be sent forward for Phase II testing.

Phase II Trials

This phase of testing usually focuses on treating one specific type of cancer and involves fewer than 100 patient participants. Phase II trials are where the effectiveness of the new drug or therapy against a particular type of cancer is evaluated. Researchers evaluate how the new treatment affects the body as a whole as well as testing the treatment's effectiveness in eradicating specific cancer cells.

Phase III Trials

In phase III trials, researchers compare the safety and effectiveness of the new drug or therapy against the safety and effectiveness of current standard therapy. These studies can include hundreds of patient participants at different treatment centers across the country. Patients who qualify for these studies are placed randomly into either the new treatment group or the standard treatment group to prevent bias from affecting the integrity of the outcome.

Protocol

Each clinical trial has its own protocol. The protocol states:

Why the research is necessary
What is to be researched
How the research will be done
The eligibility criteria, or what types of patient participants will be allowed into treatment

Eligibility Criteria

Having very specific eligibility criteria ensures that the new drug or therapy is tested on a group of people who all have the same characteristics and who can all benefit the most from a favorable outcome of the study. These criteria also help to make sure that the findings of the study reflect the actual effects of the new drug or therapy and that no outside circumstances changed the outcome. Eligibility Criteria usually include: