Clinical Trials
About Clinical Trials
Clinical trials are research studies that evaluate the safety and effectiveness of new drugs, surgical procedures, and other treatments. Before a drug or procedure is tested in a clinical trial with human beings, it will have already been evaluated extensively in laboratory and animal studies, and will have demonstrated potential usefulness. Before it can be accepted and used widely, the drug or procedure is subjected to successive stages of trials with human volunteers.
Treatment trials are designed to address specific questions about a new treatment, or a variation on an existing treatment. A treatment trial may involve a new drug, or an untested combination of drugs, or a new way of targeting drugs to the tumor. Treatment trials may also evaluate a new surgical procedure, or a new procedure for radiation. Only one new treatment will be involved in a trial.
Supportive care trials evaluate methods to reduce painful and unpleasant symptoms of the disease, or side effects of the treatment, such as depression, nausea and vomiting, and sleep disorders.
People who take part in clinical trials have an opportunity to contribute to progress in developing more effective treatment for mesothelioma. Trials are conducted by researchers who have the most up-to-date knowledge about the disease, and patient care in clinical trials is state of the art. Clinical trials are usually conducted at major medical centers and research institutions.
Phases of clinical trials
Clinical trials proceed in a series of steps, or phases, each phase building on the knowledge gained in the preceding phase.
The goal of Phase I trials is to test the safety of a new medication, and to establish safe and effective dosages. They usually involve a small number of people. Researchers study how a medication should be administered (injection, by mouth, etc), how frequently, and how much can be tolerated. Dosages are increased slowly, with careful monitoring of side effects.
Phase II trials evaluate how the drug or procedure affects the body, with the goal of learning more about its effectiveness. Phase II trials usually involve fewer than 100 subjects. Only if the drug or procedure appears to be both safe and effective, with tolerable side effects will testing move to Phase III.
Phase III trials test the new drug or procedure against the current best therapy, to learn whether it is an improvement over the current therapy. These are usually "double blind" studies, in which study patients are randomly assigned either the current therapy or the new drug or procedure, and neither patients nor research staff know which group an individual is assigned to until the study ends. Researchers feel that double blind studies reduce bias, and produce the most reliable data. Phase III studies require more patients, and are often conducted simultaneously at multiple sites around the country.
Phase IV trials are conducted on drugs and procedures already in general use, to assess their long-term safety and effectiveness. These studies tend to be large, enrolling as many as several thousand patients. They are also conducted much less frequently.
Eligibility
Because a clinical trial is structured to address specific questions about the effectiveness of the drug or procedure for a particular stage of the illness, researchers frequently limit study eligibility to patients with the specific stage. Eligibility may also be limited to patients who meet certain health criteria, to assure that they can tolerate the drug or procedure. Clinical trial listings will describe the eligibility criteria.
Benefits and Risks of Clinical Trials
For a person newly diagnosed with mesothelioma, clinical trials may offer the promise of more aggressive treatment, or a new approach to their disease. Here are some perspectives on benefits and risks prepared by the National Cancer Institute:
Benefits
- Participants have access to promising new approaches that are often not available outside the clinical trial setting.
- The approach being studied may be more effective than the standard approach.
- Participants receive regular and careful medical attention from a research team that includes doctors and other health professionals.
- Participants may be the first to benefit from the new method under study.
- Results from the study may help others in the future.
Risks
- New drugs or procedures under study are not always better than the standard care to which they are being compared.
- New treatments may have side effects or risks that doctors do not expect or that are worse than those resulting from standard care.
- Participants in randomized trials will not be able to choose the approach they receive.
- Health insurance and managed care providers may not cover all patient care costs in a study.
- Participants may be required to make more visits to the doctor than they would if they were not in the clinical trial.
Deciding whether to enter a clinical trial
A decision to enter a clinical trial is highly individual. People with mesothelioma considering a trial should discuss the trial with their healthcare providers and with loved ones. The NCI recommends raising these questions:
The study
- What is the purpose of the study?
- Why do the researchers think the approach being tested may be effective? Has it been tested before?
- Who is sponsoring the study?
- Who has reviewed and approved the study?
- What are the medical credentials and experience of the researchers and other study personnel?
- How are the study results and safety of participants being monitored?
- How long will the study last?
- How will the results be communicated?
- Possible risks and benefits
- What are the possible short-term benefits?
- What are the possible long-term benefits?
- What are the short-term risks, such as side effects?
- What are the possible long-term risks?
- What other treatment options are available?
- How do the possible risks and benefits of the trial compare with those of other options?
Participation and care
- What kinds of treatment, medical tests, or procedures will the participants have during the study? How often will they receive the treatments, tests, or procedures?
- Will treatments, tests, or procedures be painful? If so, how can the pain be controlled?
- How do the tests in the study compare with what people might receive outside the study?
- Will participants be able to take their regular medications while in the clinical trial?
- Where will the participants receive their medical care? Will they be in a hospital? If so, for how long?
- Who will be in charge of the participants' care? Will they be able to see their own doctors?
- How long will participants need to stay in the study? Will there be follow-up visits after the study?
- Personal Issues
- How could being in the study affect the participants' daily lives?
- What support is available for participants and their families?
- Can potential participants talk with people already enrolled in the study?
Cost Issues
- Will participants have to pay for any treatment, tests, or other charges? If so, what will the approximate charges be?
- What is health insurance likely to cover?
- Who can help answer questions from my insurance company or health plan?
Finding clinical trials
The National Cancer Institute (NCI), www.cancer.gov/clinicaltrials and the Mesothelioma Applied Research Foundation (MARF) www.marf.org maintain up to date lists of clinical trials, with eligibility criteria.