What is a Clinical Trial?
Clinical trials are research studies in which people help doctors find ways to improve health and cancer care. Each study tries to answer scientific questions and to find better ways to prevent, diagnose, or treat cancer.
Why are there Clinical Trials?
A clinical trial is one of the final stages of a long and careful cancer research process. Studies are done with cancer patients to find out whether promising approaches to cancer prevention, diagnosis, and treatment are safe and effective.
What are the Different Types of Clinical Trials?
Treatment trials test new treatments (like a new cancer drug, new approaches to surgery or radiation therapy, new combinations of treatment, or new methods such as gene therapy.
Prevention trials test new approaches, such as medicines, vitamins, minerals, or other supplements that doctors believe may lower the risk of a certain type of cancer. These trials look for the best way to prevent cancer in people who have never had cancer, or to prevent cancer from coming back, or a new cancer occurring in people who have already had cancer.
Screening trials test the best way to find cancer, especially in its early stages.
Quality of Life trials (also called Supportive Care trials) explore ways to improve comfort and quality of life for cancer patients.
What are the phases of Clinical Trials?
Most clinical research that involves the testing of a new drug progresses in an orderly series of steps, called phases. This allows researchers to ask and answer questions in a way that results in reliable information about the drug and protects the patients. Clinical trials are usually classified into one of three phases:
- Phase I Trials: These first studies in people evaluate how a new drug should be given (by mouth, injected into the blood, or injected into the muscle), how often, and what dosage is safe. A Phase I trial usually enrolls only a small number of patients, sometimes as few as a dozen.
- Phase II Trials: A Phase II trial continues to test the safety of the drug, and begins to evaluate how well the new drug works. Phase II trials usually focus on a particular type of cancer.
- Phase III Trials: These studies test a new drug, a new combination of drugs, or a new surgical procedure in comparison to the current standard. A participant will usually be assigned to the standard group or the new group at random (called randomization). Phase III trials often enroll large numbers of people and may be conducted at many doctors’ offices, clinics, and cancer centers.
Where Do Ideas for Clinical Trials Come From?
The ideas for clinical trials often originate in the laboratory. Researchers develop a clinical trial protocol (a plan for a trial) after laboratory studies indicate the promise of a new drug or procedure. The first trials of a particular drug or procedure are focused on safety (Phase I), and later trials focus on whether the drug or procedure is effective (Phase II or Phase III).
What is a Protocol?
Every trial has a person in charge, usually a doctor, who is called the protocol chair or principal investigator. Phase I and Phase II studies generally refer to the person in charge as the principal investigator. Phase III studies generally have a protocol chair, under whose direction multiple principal investigators carry out the protocol in participating sites. The protocol chair or principal investigator prepares a plan for the study, called a protocol. The protocol explains what the study will do, how it will be carried out, and why each part of the study is necessary. For example, the protocol includes:
- The reason for doing the study
- How many people will be in the study
- Who is eligible to participate in the study
- What study drugs the participants will take, if any
- What medical tests they will have, if any, and how often
- What information will be gathered
Every doctor or research center that takes part in the trial uses the same protocol. This ensures that patients are treated identically, and that information from all the participating centers (if there are more than one) can be combined and compared.
Who Sponsors Clinical Trials?
Clinical trials are sponsored by organizations or individuals who are seeking better treatments for cancer or better ways to prevent or detect cancer.
Individual physicians at cancer centers and other medical institutions can sponsor clinical trials themselves.
The National Cancer Institute (NCI) sponsors a large number of clinical trials. The NCI has a number of programs designed to make clinical trials widely available in the United States. Thousands of investigators at over a thousand sites participate in various aspects of NCI’s clinical trials program. These include:
- Cancer Center Program: About 60 research-oriented institutions have been designated as an NCI Comprehensive or Clinical Cancer Center for their scientific excellence. The centers are key partners in the NCI’s efforts to bring the benefits of clinical research directly to you. Located throughout the country, they play an important role in cancer research, delivery of the highest quality cancer care, and outreach and education for the public and professionals.
- Cooperative Clinical Trials Program: This program brings together groups of researchers, cancer centers, and community physicians into a national NCI-supported network. The network consists of a number of Cooperative Groups that seek to define the key unanswered questions in cancer and then conduct high-quality clinical trials at many sites around the country to answer these questions. The Cooperative Groups enroll about 20,000 new patients in treatment trials each year. Important Phase III trials run by the Cooperative Groups help establish the state-of-the-art for cancer therapy. Additionally, the Groups perform large cancer prevention trials.
- The National Cancer Institute Community Oncology Research Program: This program makes clinical trials available in a large number of local communities in the United States by linking community physicians with researchers in cancer centers. Local hospitals throughout the country affiliate with a cancer center or a Community Oncology Research Program. This enables doctors to offer people participation in clinical trials more easily, without having to travel long distances or leave their usual caregivers. Several of these programs focus on encouraging minority populations to participate in trials. The Nevada Cancer Research Foundation (NCRF) NCI Community Oncology Research Program has been funded by the National Cancer Institute since 1994. The LHMCC has been a part of a community clinical oncology program since 2007.
Should I take part in a Clinical Trial?
Only you can make the decision about whether or not to participate in a clinical trial. Before making your decision, you should:
- Learn as much as possible about your disease and the trials that are available to you.
- Talk to your doctor/or nurse, family members and friends to help you determine what is right for you.
What are the potential risks and benefits of Clinical Trials?
Potential benefits include:
- Health care provided by leading physicians in the field of cancer research.
- Access to new drugs and interventions before they are widely available.
- Close monitoring of your health care and any side effects.
- A more active role in your own health care.
- If the approach being studied is found to be helpful, you may be among the first to benefit.
- An opportunity to make a valuable contribution to cancer research.
The potential risks include:
- New drugs and procedures may have side effects or risks unknown to the doctors.
- New drugs and procedures may be ineffective, or less effective, than current approaches.
- Even if a new approach has benefits, it may not work for you.
How are Participants Protected?
The government has a system designed to protect human research subjects. Before a government-funded clinical trial can begin, the trial plan (also called a protocol) must be approved. During the trial, review committees make sure that the plan is being followed and participants are being protected.
Regulations require the researchers performing studies to thoroughly inform patients about a study’s treatment, tests, their possible benefits, and potential risks before a patient decides whether or not to participate in any study. This process is called informed consent.
The NCRF Central Oncology Institutional Review Board (IRB) approves all local research studies. The IRB includes both scientific and non-scientific members and is dedicated to the integrity of the research goals and objectives as well as protecting the rights of all participants.
Assuring that the process to enroll study participants includes informed consent on behalf of patients is a major responsibility of the IRB. Anyone participating in a research protocol has a right to understand the pros and cons of participation. The IRB reviews the informed consent document for each patient enrolled in a NCRF IRB-approved study.
What is Informed Consent?
Informed consent is a process in which you learn the key facts about a clinical trial before you decide whether or not to participate. In addition to talking about these facts with the research doctor or nurse, the facts will be included in a written consent form that you can take home to read and discuss. The consent form will include details about:
- The study approach
- The intervention given in the trial
- The possible risks and benefits
- The tests you may have
Don’t hesitate to ask questions until you have all the information you need. While informed consent begins before you agree to participate in a trail, you should feel free to ask the health care team any questions you have at any point. Informed consent continues as long as you are in the study. You can change your mind and leave the study whenever you want – before the study starts or at any time during the study or follow-up period.
Could I receive a Placebo?
In treatment trials involving people who have cancer, placebos (“dummy” pills that contain no active ingredient) are rarely used.
Many treatment trials are designed to compare a new treatment with a standard treatment, which is the best treatment currently known for a cancer based on results of past research. In these studies, patients are randomly assigned to one group or another.
When no standard treatment exists for a cancer, a study may compare a new treatment with a placebo. However, you will be told about this possibility during informed consent, before you decide whether or not to take part in the study.
What Happens During a Clinical Trial?
If you decide to participate in a clinical trial, you will work with a research team. Team members may include doctors, nurses, social workers, dietitians and other health care professionals. They will provide your care, monitor your health carefully, and give you specific instructions about the study.
Participating in a trial may mean that you might have more tests and doctor visits than you would if you weren’t in the study. Team members also may continue to stay in contact with you after the trial ends. To make the trial results as reliable as possible, it is important for participants to follow the research team’s instructions. That means attending all doctor visits and having tests, taking medicines on time, and completing logs or answering questionnaires.
What happens when a Clinical Trial is over?
After a Phase I or Phase II trial is completed, the researchers look carefully at the data collected during the trial to decide whether to:
- Move to the next trial with the treatment, or
- Stop testing the treatment because it is not safe or effective
When a Phase III trial comes to an end, the researchers must look at the data and decide if the results have medical importance. When the analysis of a Phase I, Phase II, or Phase III trial is complete, the researchers will inform the medical community and the public of the study results.
In most cases, the results of trials are published in scientific or medical journals. To find out if the results of a study you participated in was published, ask the doctor or nurse in charge of your treatment.
Most medical and scientific journals have in place a process of peer review, in which experts critique the report before it is published, to make sure that the analysis and conclusions are sound. Particularly important results are likely to be featured by the print or electronic media, and widely discussed at scientific meetings and by patient advocacy groups. Once an intervention is proven safe and effective in a clinical trial, it may become the new standard of practice. In this way the development of better interventions for prevention, for treatment, or for detection and diagnosis is an ongoing, continuous process that builds progressively on itself to improve the quality of cancer care and prevention available to all.