What Are Clinical Trials

8/13/2019 What Are Clinical Trials

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What Are Clinical Trials?

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The following data has largely adapted from Taking part in clinical trials, what cancer patientsneed to know, published by the National Cancer Institute.

Clinical trials, also called cancer treatment or research studies, test new treatments andtherapies in people with cancer. The goal of these trials is to find better ways totherapies and treatments for cancer. Clinical trials in general, seek to answer specificscientific questions to find better ways to prevent, detect, and treat diseases as well asto improve overall care for people with disease. Clinical trials may test many types oftreatment such as new drugs, new approaches to surgery or radiation therapy, newcombinations of treatments, or new methods such as gene therapy. The search for newtreatments begins in the laboratory, where scientists first develop and test new ideas. If

an approach seems promising, the next step may be testing a treatment in animals tosee how it affects a particular cancer in a living being and whether it has harmful effects.Many different mammals are used in this type of research, from rats to monkeys. One ofthe most common models for oral cancer is the hamster cheek pouch model. Of course,treatments that work well in the lab or in animals do not always work well in people.Once a particular treatment modality proves effective in an animal model, it may thenprogress to human clinical trials. In these trials researchers will find out whetherpromising treatments are as safe and effective as they were in the animal studies.
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Types of Clinical TrialsThere are several different types of cancer clinical trials. Each type of trial is designed toanswer different research questions:

1. Prevention trials:

What approaches can prevent a specific type of cancer from developing inpeople who have not previously had cancer?2. Early-detection/screening trials:

What are new ways of finding cancer in people before they have any symptoms?3. Diagnostic trials:

How can new tests or procedures identify a suspected cancer earlier or moreaccurately?

4. Genetics trials:Can gene-transfer therapy be used to treat cancer?

5. Treatment trials:What new treatment approaches can help people who have cancer?

What is the most effective treatment for people who have cancer?6. Quality-of life and supportive care trials:What kinds of interventions can improve the comfort and quality of life of peoplewho have cancer?

7. What Happens in a Clinical Trial?In a clinical trial, patients receive treatments which are new and innovative,though not completely proven to work or be risk free. Doctors carry out researchon how the treatment affects the patients, and whether the desired results occur.While clinical trials have risks for the people who participate, each study alsotakes steps to protect patients.

What Is It Like To Receive Treatment in a Clinical Trial?When you take part in a clinical trial, you receive your treatment in a cancer center,hospital, clinic, and/or doctors office, which has qualified to become part of the stud y.These doctors and institutions have had to submit a rigorous protocol for approvalbefore they may participate in a clinical trial. Doctors, nurses, social workers, and otherhealth professionals may be part of your treatment team. They will follow your progressclosely. You may have more tests and doctor visits than you would if you were nottaking part in a study. You will follow a treatment plan your doctor prescribes, and youmay also have other responsibilities such as keeping a log or filling out forms about yourhealth. Many studies continue to check on patients even after their treatment is over,and it is not uncommon to have mandatory follow-up visits well after treatment hasstopped. With a new therapy or treatment, it may be some time before completeunderstanding of the effects are completely understood.


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How Is the Research Carried Out?

In clinical trials, both research concerns and patient well being are important. To helpprotect patients and produce sound results, research with people is carried outaccording to strict scientific and ethical principles.

These include:

1. Each clinical trial has an action plan (protocol) that explains how it will work.The studys investigator, usually a doctor, prepares an action plan for the study.The protocol, explains what will be done in the study and why. It outlines howmany people will participate, what common characteristics they have (such asage, extent of disease etc.), what medical tests they will receive and how often,and the expected outcome of the protocol. Exactly the same protocol is used byeach doctor that takes part in the trial. For patient safety, each protocol must beapproved by the organization that sponsors the study (such as the National

Cancer Institute) and the Institutional Review Board (IRB) at each hospital orother study site. This board, which includes lay people, clergy, and healthprofessionals, reviews the protocol to try to be sure that the research will notexpose patients to extreme or unethical risks.

2. Each study enrolls people who are alike in key ways. Each studys protocoldescribes the characteristics that all patients in the study must have. Calledeligibility criteria, these guidelines differ from study to study, depending on theresearch purpose. They may include age, gender, the type and stage of cancer,and whether cancer patients who have had prior cancer treatment and whattypes of treatments those have been, or if people who have other healthproblems can take part. Using eligibility criteria is an important principle of

medical research that helps produce reliable results. During a study, they helpprotect patient safety, so that people who are likely to be harmed by study drugsor other treatments are not exposed to the risk. After results are in, they also helpdoctors know which patient groups will benefit if the new treatment being studiedis proven to work. For instance, a new treatment may work for one type of cancerbut not for another, or it may be more effective for men than women.

3. Cancer clinical trials include research at different phases. Each phase answersdifferent questions about the new treatment.

Phase I trials are the first step in testing a new treatment in humans. In these studies,researchers look for the best way to give a new treatment (e.g., by mouth, IV drip, orinjection? how many times a day?). They also try to find out if and how the treatmentcan be given safely (e.g., best dose?); and they watch for any harmful side effects.Because less is known about the possible risks and benefits in Phase I, these studiesusually include only a limited number of patients, between 15 and 30, who would not behelped by other existing, known treatment modalities.Phase II trials continue to test thesafety of the new treatment, and begin to evaluate how well it works against a specifictype of cancer. As in Phase I, only a small number of people (less than 100) take part.In general, these participants have been treated with chemotherapy, surgery or


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radiation, but treatment has not been effective. It is important to remember that when aphase II trial begins, it is not yet known if the agent tested works against the specificcancer being studied. Unpredictable side effects can also occur in these trials.

Phase III trials focus on how a new treatment compares to standard treatment,

(treatment currently accepted and most widely used). Researchers want to learnwhether the new treatment is better than, the same as, or worse than the standardtreatment. In most cases, studies move into Phase III testing only after a treatmentshows promise in Phases I and II.

Phase III trials may include hundreds to thousands of people, a many clinical centers,ranging from people newly diagnosed with cancer to people with extensive disease. InPhase III trials, people are assigned at random to receive either the new treatment orstandard treatment.

Phase IV trials are used to further evaluate the long-term safety and effectiveness of a

treatment. Less common than phase I, II, and III trials, phase IV trials usually take placeafter the new treatment has been approved for standard use.

Researchers assign patients by chance either to a group taking the new treatment(called the treatment group) or to a group taking standard treatment (called the controlgroup). This method, called randomization, helps avoid bias: having the studys resultsaffected by human choices or other factors not related to the treatments being tested. Insome studies, researchers do not tell the patient whether he or she is in the treatment orcontrol group (called a single blind study). This approach is another way to avoid bias,because when people know what drug they are taking, it might change the way theyreact. For instance, patients who knew they were taking the new treatment might expect

it to work better and report hopeful signs because they want to believe they are gettingwell. This could bias the study by making results look better than they really were.

How Are Patients Protected?

The Informed Consent Process Informed consent, as a legal, regulatory, and ethicalconcept, is an integral part of research. In clinical trials, informed consent is the processof providing all relevant information about the trials purpose, risks, benefits,alternatives, and procedures to a potential participant, who then, consistent with his orher own interests and circumstances, makes an informed decision about whether or notto participate. The informed consent process provides people with ongoing explanations

that will help them make educated decisions about whether to begin or to continueparticipation in a clinical trial. The process does not end with the signing of informedconsent documents. If new benefits, risks or side effects are discovered during the trial,researchers must inform participants. Participants are encouraged to ask questions atany time. Before agreeing to take part in a clinical trial, participants have the right tolearn everything that is involved in the trial - including all details about treatment, tests,and possible risks and benefits. The information has to be in a format and language thatcan be easily understood.


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Why Are Clinical Trials Important?

First, cancer affects us all, whether we have it, care about someone who does, or worryabout getting it in the future. Clinical trials contribute to our knowledge base, andprogress against cancer. If a new treatment proves effective in a study, it may become a

new standard treatment that can help many patients. Many of todays most effectivestandard treatments are based on previous clinical trial study results. Examples includetreatments for breast, colon, rectal, and childhood cancers. Clinical trials may alsoanswer important scientific questions and suggest future research directions. Becauseof progress made through clinical trials, many people treated for cancer are now livinglonger. Second, the patients who take part may be helped personally by thetreatment(s) they receive. They get up-to-date care from cancer experts, and theyreceive either a new treatment being tested or the best available standard treatment fortheir cancer. Of course, there is no guarantee that a new treatment being tested or astandard treatment will produce good results. New treatments also may have unknownrisks. But if a new treatment proves effective or more effective than standard treatment,

study patients who receive it may be among the first to benefit. Some patients receiveonly standard treatment and benefit from it. In the past, clinical trials were sometimesseen as a last resort for people who had no other treatment choices. Today, patientswith common cancers often choose to receive their first treatment in a clinical trial.

Clinical Trials: Weighing the Pros and Cons

While a clinical trial is a good choice for some people, this treatment option has bothpossible benefits and drawbacks.

Possible Benefits

Clinical trials offer high-quality cancer care. If you are in a study and do not receive thenew treatment being tested, you will receive the best standard treatment. This may beas good as, or better than, the new approach. If a new treatment approach is proven towork and you are taking it, you may be among the first to benefit. By looking at the prosand cons of clinical trials and your other treatment choices, you are taking an active rolein a decision that affects your life. You have the chance to help others and improvecancer treatment.

Possible DrawbacksNew treatments under study may not be better than, or even as good as standard care.They may have side effects that doctors do not expect or that are worse than those ofstandard treatment. Even if a new treatment has benefits, it may not work for you. Evenstandard treatments, proven effective for many people, do not help everyone. If youreceive standard treatment instead of the new treatment being tested, it many not be aseffective as the new approach.Health insurance and managed care providers do notalways cover all patient care costs in a study. What they cover varies by plan and bystudy. To find out in advance what costs are likely to be paid in your case, talk to thedoctor, nurse or social worker from the study.


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What Are Clinical Trials

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