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This list of frequently asked questions is a starting point for understanding clinical trials for brain tumors.
- What is a clinical trial?
- What are the major phases of a clinical trial and what do they mean?
- What are the different types of clinical trials?
- Who conducts clinical trials?
- What should I consider before participating in a clinical trial?
- What happens during a clinical trial?
- What is informed consent?
- Who pays for clinical trials?
- How do I get access to clinical trials?
- What are some topics to discuss when I contact the clinical trial coordinator?
- What factors should I consider before participating in a clinical trial?
- What is the Karnofsky scale?
- What is a placebo?
- What do I do when I am done with the trial, but still want to use the treatment (or drug)?
- What is the "Institutional Review Board"?
- What is the difference between "FDA approved," "experimental" and "off-label"?
- How is the safety of the participant protected?
- Where can I get more information about clinical trials for brain tumors?
1. What is a clinical trial?
According to the National Cancer Institute there are a number of definitions for clinical trial, however, in general they are considered to be biomedical or health-related research studies in human beings that follow a pre-defined protocol.
For individuals with brain tumors and other life-threatening illnesses, one treatment option may be enrollment in a clinical trial. In addition to being an avenue for providing high-quality health care, clinical trials answer questions about the effectiveness of promising new treatments. They are a method designed to scientifically determine the effectiveness of various treatment regimens. Clinical trials for brain tumors most often evaluate chemotherapy, radiation therapy, or biologic therapy. These studies consist of three phases used in the evaluation of investigational drugs that may have therapeutic indications for patients.
Phase I: Determination of safe drug doses and/or schedules of a new drug when given to patients. The treatment has not been evaluated in people with brain tumors before. The main goal is to determine what is the safe dose of medication and what side effects the person experiences. The treatment being studied is considered promising for treating brain tumors, but evaluating response is not the primary goal of the study. Patients get a specific dose of drug(s) and are carefully observed for side effects.
Phase II: Determination of therapeutic efficacy. The safe dose of medication has already been established, and the researcher wants to determine whether the treatment shrinks the tumor and extends the person’s survival.
Phase III: Comparison of the drug to an existing, effective standard therapy. In this phase the person can receive either the experimental treatment or the standard treatment. A computer randomly assigns the person to a treatment. This process is called randomization. The researcher does not know which treatment is best, thereby preventing bias in determining which treatment the person will receive. In this way, the treatment can be compared directly to a standard treatment to determine which is a better approach to therapy.
Treatment Trials: These trials test experimental treatment, new combinations of drugs, or new approaches to surgery or radiation therapy.
Prevention Trials: These trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicine, vaccines, minerals, or lifestyle changes.
Diagnostic Trials: These trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
Screening Trials: These trials test the best way to detect certain diseases or health conditions.
Quality of Life Trials (or Supportive Care Trials): These trials explore ways to improve comfort and the quality of life for individuals with chronic illness.
4. Who conducts clinical trials?
Clinical trials are conducted by various institutions and groups. Each clinical trial follows a treatment plan or "protocol." These protocols are defined by the researchers initiating the clinical trial and undergo close scrutiny at each institution or hospital by an Institutional Review Board (IRB). The IRB reviews a clinical trial to be sure it is designed with safeguards to protect people who participate and to prevent significant risks.
The various groups that may initiate a clinical trial include
Institutions: Individual institutions or hospitals conduct studies of various drugs. The individual or group of researchers at that institution may first try a treatment in the research laboratory and then proceed to testing with people with brain tumors. Institutional trials may be Phase I, II, or III.
National Cancer Institute Sponsored CNS Consortia: These are groups of specialized brain tumor centers from around the United States that receive funding to test innovative new treatments for brain tumors. These groups test new treatments in Phase I and II testing.
National Cooperative Groups: These groups consist of both university and community hospitals throughout the United States. The goal is to test new treatments for both efficacy and the ability to be done in a community setting. National Cooperative Groups include The Eastern Cooperative Oncology Group (ECOG); The Southwestern Oncology Group (SWOG); The Radiation Therapy Oncology Group (RTOG); and the North Central Cancer Treatment Group (NCCTG). Pediatric Groups include the Pediatric Oncology Group (POG) and the Children’s Cancer Group (CCG). These groups test treatments in Phase II and III testing.
Pharmaceutical Sponsored Multi-Institutional Trials: Individual pharmaceutical companies often form consortia of hospitals to investigate a drug that they have developed. These trials can be Phase I, II, or III depending on the level of knowledge of that drug for brain tumors.
5. What should I consider before participating in a clinical trial?
People choose to take part in clinical trials for various reasons, whether to try an innovative treatment, to find hope for a cure, to attempt to feel better, or to assist in helping others in the future. If you are thinking about participating in a clinical trial, there are multiple trials from which to choose. Which trial is appropriate for you depends on several factors, including what type or grade of tumor you have when you were diagnosed and what treatments you have received and how well you are functioning. Most trials have specific restrictions based on these criteria. Use all the resources that you can to assist you in your decision. When deciding, you should ask several questions about the particular clinical trial:
- Who is conducting the study? Is it the individual institution or is it a part of a national trial?
- At what institutions is the study being conducted? Is there an institution closer to home?
- Does the center have a designated neuro-oncology (or brain tumor) program?
- What is the phase of the study? Has it been tested on people with brain tumors before?
- What tests, treatments, hospital stay, and commitment of time does the study involve?
- What other choices are available? How does the study treatment compare?
- What potential side effects may occur and how might my day-to-day life be affected?
- What tests will I need during the trial and how often will they be done?
- How will my participation affect my ability to participate in future clinical trials involving the same or other drugs?
- What does the treatment cost and is any part provided for free?
Obtaining answers to all of these questions can help you determine if the treatment is right for you. There are no bad questions when learning about your treatment options. Learning as much as you can before starting a treatment will help you to be prepared for what occurs.
6. What happens during a clinical trial?
If you participate, the first step involves giving informed consent to indicate that you fully understand the trial’s risks and benefits. You will then be asked to sign a consent form. This does not mean that you cannot leave the trial, but only that you understand the protocol. You may elect to leave a clinical trial at any point in the process. During the course of the clinical trial, you will be evaluated closely and required to report symptoms that you experience. This may require extra physical examinations, tests, or filling out forms. The informed consent form will outline this for you.
7. What is informed consent?
Informed consent means that if you elect to participate in the trial, you fully understand the risks and benefits of the treatment and freely elect to take part. Informed consent for clinical trials is just one part of a larger system in place to safeguard people who want to help researchers evaluate new practices that may improve treatment, supportive care, screening, and prevention, while perhaps benefiting from these new methods. This system ensures that clinical trials are conducted ethically, without undue risk to participants. Even after you give your consent, you can leave a clinical trial at any time.
Source: National Cancer Institute
For additional information on informed consent documents, please visit the National Cancer Institute website at http://www.cancer.gov/clinicaltrials/conducting/informed-consent-guide/page3
8. Who pays for clinical trials?
According to the National Cancer Institute, some clinical studies have a sponsor, for example a pharmaceutical company or a biotechnology company. These sponsors may pay for the new drug being tested. In some cases they can also help cover the costs of hospital and/or doctor visits, x-rays, or other procedures. In most cases, however, you or your insurance company will most likely be responsible for these additional costs. If you decide to participate in a clinical trial the doctor, nurse or clinical trial coordinator will explain the options to you in greater detail.
It is also important to consider any additional travel or lodging costs if you will be receiving treatment at a center far from home as well as direct treatment-related costs.
STEP 1: Learn as much about your disease and diagnosis as you possibly can.
Know the name, grade, and size of the tumor, tumor location(s), and the standard treatment(s) for your diagnosis. If you have a recurrence or have already received treatment, you should know your previous treatment(s) including drug names, type of radiation treatment(s), dates and number of treatments, and the location and extent of the recurrence.
STEP 2: Type or write down and save your diagnosis and treatment information. Keep it accessible.
Remember to always have this information handy when inquiring about a clinical trial. Practice summarizing your diagnosis; give no irrelevant information, just the facts. You should be able to do this in a few minutes, similar to how one doctor might report your case to another doctor.
STEP 3: Request help if you need it.
If you don’t have all the information listed in step 1, ask your doctor’s staff to assist you with the details of your diagnosis. The information in your medical record belongs to you.
STEP 4: Let your doctor know that you are investigating your clinical trial option.
Physicians, especially your oncologist, are extremely busy and may not have the time or staff available to explore this option with you. However, let your doctor know that you welcome advice while you are gathering information. Be sure your doctor understands that you are only in the information-gathering stage and that, once you determine the trials for which you may be eligible, you will you want to discuss your potential choices.
STEP 5: Find clinical trial listings on the internet or by telephone.
Look at the trials listed on the following websites or call 800.4.CANCER (422.6237)
Please also review the following points:
- he trial objective (what scientists are trying to learn from conducting this study)
- The locations where the trial will be conducted
- The requirements (called, "patient eligibility criteria") used to determine which patients can be in the trial. Examples include age, extent of disease, and type(s) of prior treatment received.
STEP 6: Compare the trial eligibility criteria with your diagnosis.
Select trials that seem to match. Narrow the selection down to two or three before you seek your doctor’s advice. Avoid presenting your doctor with a stack of trials. If you need help in narrowing the choice, a cancer patient advocacy organization can assist you.
10. What are some topics to discuss when I contact the clinical trial coordinator?
After you have reviewed a trial’s eligibility criteria, call to find out whether you are in fact eligible for the trial. Your doctor can also call the trial site for you, although this is not as necessary while you are in the process of gathering information.
When you call the clinical trial coordinator, ask to speak to the protocol nurse or research assistant.
Ask the research assistant or protocol nurse the following questions:
- Is the trial still recruiting brain tumor patients? (If the trial is close to your home, you can ask if there are other trials at that location that you may be eligible to enter.)
- Am I eligible for the trial? (Be prepared to present a brief summary of your tumor type, age, treatment, etc.)
- What is the trial objective?
- How many brain tumor patients have been recruited thus far? How many are being recruited total?
- May I see the informed consent document?
If the trial is still open and you are eligible, you may express your interest in entering the trial. You should now consult your family and physician before making your final decision.
11. What factors should I consider before participating in a clinical trial?
Consider how far you are willing to travel from your home. Many patients prefer to enter a trial that is being conducted within an hour from their home. Often, the same trial is conducted in more than one location.
You may also want to consider the following in you decision-making process:
- Will my health insurance pay for my care if I enter a trial?
- Should I do it if my doctor disagrees?
There is support available to patients in clinical trials. For example, trials conducted by the National Cancer Institute at the facility in Bethesda, MD only are free and include reimbursement for some travel expenses.
Lastly, the staff in the Food and Drug Administration's (FDA) Office of Special Health Issues is skilled and knowledgeable on this subject and is available to help you. The email address is OSHI@oc.fda.gov.
12. What is the Karnofsky scale?
The Karnofsky scale is a tool used to measure a patient's ability to function and carry on normal activities, cognitive function, and overall well-being. The scores can range from 0 (nonfunctional) to 100 (completely normal functioning). This measurement is often used as one of the eligibility criterion for a particular clinical trial.
Karnofsky Performance Status Chart:
|100||Normal: no complaints, no evidence of disease|
|90||Able to carry on normal activity; minor symptoms|
|80||Normal activity with effort; some symptoms|
|70||Cares for self; unable to carry on normal activities|
|60||Requires occasional assistance; cares for most needs|
|50||Requires considerable assistance and frequent care|
|40||Disabled: requires special care and assistance|
|30||Severely disabled: hospitalized but death not imminent|
|20||Very sick: active supportive care needed|
|10||Moribund: fatal processes are progressing rapidly|
Source: Cleveland Clinic
You will need to find out whether you meet the eligibility criteria for the trial(s) you are interested in joining. Criteria may include things like age, tumor type, grade of tumor, overall health, previous treatment, length of time since you last received treatment, etc.
13. What is a placebo?
In a clinical trial, a group of human volunteers receiving the investigational therapy are compared with another group that receives either the standard treatment or a placebo. Placebos are any synthetic treatment that has no therapeutic benefit. This allows the researchers to compare the effect of the treatment to those not receiving the studied treatment in otherwise similar patients. When the control group is given the standard treatment, researchers are able to determine whether the experimental treatment provides a better outcome than what is already available.
The clinical trial setting helps ensure that risks are minimized because the research protocol, the set of rules by which the clinical trial is conducted, has been scrutinized by the FDA and a local ethics committee called an Institutional Review Board.
Information adapted from www.fda.gov – experimental treatments article
14. What do I do when I am done with the trial, but still want to use the treatment (or drug)?
After a successful clinical trial, the FDA can approve a drug for use for one or more cancers. "Off-label" refers to using an FDA approved drug for a cancer other than that for which it was approved. A patient with advanced disease or with no approved treatment or clinical trial options can attempt to get access to a new, unapproved drug outside of participating in a clinical trial. Access to a drug outside of a clinical trial prior to FDA approval is commonly referred to as "compassionate use."
Source: National Cancer Institute
15. What is the "Institutional Review Board"?
Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
Source: National Cancer Institute
FDA approved: Official permission granted by the FDA allowing a drug manufacturer to market a new prescription drug for a specific health benefit. (Source: www.johnshopkinshealthalerts.com)
Experimental (multiple definitions): A new or newly discovered drug (medicine) that is still being developed, a drug that is not FDA licensed for use in humans or as a treatment for a particular condition, and/or new drug that is being tested as a treatment for a specific condition and has not yet been approved for use.
Off-Label: The legal use of a prescription drug to treat a disease or condition for which the drug has not been approved by the FDA for that particular disease or condition. (Source: National Cancer Institute)
17. How is the safety of the participant protected?
The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built-in safeguards to protect participants. Clinical trials follow a carefully controlled protocol, which is a study plan that describes in detail what researchers will do in the study.
As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. The individual participants' names will remain secret and will never be mentioned in the reports.
For more information on how clinical trials are monitored refer back to question 15.
Source: National Cancer Institute