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Ed Forum 2016

Understanding Clinical Trials

What are clinical trials?

Clinical trials are research studies in human volunteers, with the goal of developing better ways to prevent, screen for, diagnose, or treat a disease. Cancer clinical trials may include new drugs, new surgical or radiation therapy techniques, new combinations of treatments, and revolutionary methods such as gene therapy.

Why are clinical trials necessary?

It has been found that most effective cancer treatments are the direct result of clinical trials. Clinical trials can help answer questions about the safety and effectiveness of new therapies, optimal dosage and frequency of dosage, side effects, duration of remission, and comparison with other therapies already in use. Results from clinical trials may also suggest future research directions.

Why should I enter a clinical trial?

People who take part in cancer clinical trials have an opportunity to contribute to knowledge and progress in cancer treatment. They receive up-to-date care from cancer experts and undergo either a new promising treatment or the best available standard treatment for their cancer. Whether one should participate in a clinical trial is a personal decision which should be made only with input from your physicians.

How are clinical trials carried out?

Research with humans is conducted according to strict scientific and ethical principles. Patients who join clinical trials are given either some of the best treatment currently available or the chance to receive a new treatment under consideration. Placebos (so-called sugar pills) are rarely used in cancer clinical trials and are never used in place of treatment. Every clinical trial has a “protocol”, a detailed action plan that acts as a recipe for conducting the trial. The same protocol is used by every physician or research center taking part in the trial. The “outcome” or “endpoint” of the study is its specific goal or purpose.

Each trial enrolls a certain number of eligible volunteer patients who may be alike in key ways – type of cancer, age, gender, history of prior treatments, current health status, etc. – these are called “inclusion criteria.” Even though patients may meet inclusion criteria, they may not be eligible for a particular trial because they have "exclusion criteria," namely other medical conditions or prior treatments that exclude them from participation in the trial. Clinical trials take place in doctors’ offices, cancer centers, other medical centers, community hospitals and clinics, and veterans' and military hospitals in cities and towns across the U.S. and in other countries.

What is informed consent?

Informed consent is a process by which patients learn the important facts about a clinical trial to help them decide whether to participate. This information includes details about the purpose of the study, the tests and other procedures used in the study, and the possible risks and benefits. Patients receive a written consent form explaining the study. The informed consent process continues for the duration of the study. If new benefits, risks, or side effects are discovered during the study, researchers must notify the participants. Signing the written consent form does not mean that patients are obligated to remain in the study for its duration – they may withdraw from the study at any time.

Who pays for the cost of clinical trials?

The trial sponsor, which may be a government entity, a research institution, a business such as a pharmaceutical company, or any combination thereof, usually pays for the cost of the treatment being studied and for costs associated with any special testing or extra doctor visits that may be required. Travel costs and routine costs (which are the usual costs of medical care, such as doctor visits, hospital stay, laboratory tests, X-rays, etc., that you would receive whether or not you were participating in a clinical trial), may not be covered by your health plan while you are in a trial.

In the U.S. certain states now have laws that require insurers to pay for the costs of routine care that occur doing the course of a clinical trial. Medicare may also cover some of the costs connected with a clinical trial. Contact your health insurer or the clinical trial research coordinator, research nurse, or social worker for assistance in determining what costs will be covered and what your financial responsibility will be. Because a lack of coverage for some of these costs can keep people from enrolling in trials, the National Cancer Institute is working with major health plans to find solutions.

What are the different “phases” of clinical trials?

Clinical trials are usually conducted in a series of steps, called phases.

Phase I trials are the first step in testing a new treatment in humans. Researchers evaluate what dose is safe, how a new agent should be given (by mouth, injection into a vein, injection into a muscle) and how often. Researchers watch for harmful side effects. The dose of the new therapy or technique is increased a little at a time. The highest dose with an acceptable level of side effects is determined to be appropriate for further testing. Phase I trials usually include only a limited number of patients and generally are carried out at large academic medical centers.

Phase II trials attempt to determine whether the new agent or technique works for the specific cancer and continue to study its safety and effectiveness.

Phase III trials compare the treatment outcomes of patients taking the new therapy with results of people taking standard treatment. Participants are randomly assigned to the standard (also called control) group or to the new treatment group. This method, called randomization, helps to avoid bias and ensures that human choices or other factors do not affect the study’s results. Randomized trials can be set up as “single-blind” or “double-blind” studies. In single-blind studies, patients do not know whether they are receiving the new treatment or the standard treatment; in double-blind studies, research coordinators know which treatment is being administered, but neither the patient nor the clinical physicians and nurses know. In most cases, studies move into Phase III testing only after they have shown promise in Phases I and II. Phase III trials often include large numbers of participants.

Phase IV trials occur after a treatment has been approved and is being marketed. The drug’s maker may study it further to evaluate the side effects, risks, and benefits over a longer period of time and in a larger number of people than in Phase III clinical trials.

Questions to Ask your Doctor about Clinical Trials

If you are considering participating in a Clinical Trial for treatment, the National Institutes of Health (NIH)/National Cancer Institute has developed a helpful set of questions which you may want to ask your doctor. These can be found here.

Where Can I Find Clinical Trials for WM

Clinical trials for WM can be found by entering Waldenstrom’s macroglobulinemia in the “Search Studies” block at www.clinicaltrials.gov.