Cancer Research: Information from a Patient / Caregiver Perspective |
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 | Phase I Oncology Clinical Trial Questions and AnswersThis information on this page is compiled from both personal experience and research, in large part from the National Cancer Institute (US) website, which offers more comprehensive information on this subject than is available on the Health Canada website. What is a Phase 1 cancer clinical trial? What is a Phase 1 cancer clinical trial?A Phase 1 cancer clinical trial is the first time an experimental cancer treatment is used on human beings. What is the purpose of a Phase 1 cancer clinical trial?The purpose of Phase 1 cancer clinical trial is to determine the maximum dosage of an experimental treatment that a human body can tolerate (maximum tolerated dosage = "MTD"). Why do researchers need to know the Maximum Tolerated Dosage (MTD)?In the development process, it is necessary to determine the dosage of the experimental treatment that is ‘safe’ for use on humans. New cancer fighting treatments are developed in laboratories. Researchers perform computer simulations and then test various dosages and methods of administering the new treatment on live animals. The next step is to use the experimental treatment on live human beings. Researchers have a good idea how the various dosages of the new treatment will be tolerated by a human body; but a ‘good guess’ is not acceptable when allowing a new treatment for use on the general public. The maximum dosage of the experimental treatment, that is safe to use on a human being, is determined by the Phase 1 study. There is a distinct possibility that a lethal dose could be administered. The safe dosage, also determined by the Phase 1 study, is then used in a Phase 2 study to see if the new treatment is an effective anti-cancer weapon. If the new treatment is found by the Phase 2 study, to be useful in the fight against cancer, then it is compared, in a Phase 3 study, to treatments currently in use. Who is used as the human test subject in a Phase 1 cancer clinical trial?Only cancer patients, who have been declared “terminally-ill” by a recognized oncologist, can volunteer to participate as test subjects for a Phase 1 clinical trial. For these patients, all possible avenues for cure or containment of their cancer have been exhausted. Not all people who are terminally-ill with cancer can be accepted into a Phase 1 study. The cancer patient’s body must meet a specified level of adequate organ function before entering the study. The volunteer must agree to provide numerous samples for laboratory analysis throughout their entire association with the Phase 1 trial. The Phase 1 volunteer must be living in very close proximity to the research facility conducting the experiments. No compensation for any out of pocket expenses is given. The volunteer must donate their time and their resources to a Phase 1 clinical study. How is a Phase 1 cancer clinical trial structured?A typical Phase 1 study requires 20 to 40 terminally-ill people, divided into cohorts. Each cohort consists of a small group of 2 to 4 volunteers. The first cohort is given a dosage of the experimental treatment that laboratory animals were able to tolerate. If the members of that cohort show no ill effects then another cohort is recruited and the new cohort is given a higher dosage of the treatment. The volunteers in each specific cohort are given exactly the same dose of the experimental treatment throughout their entire association with the Phase 1 study. Escalating dosages of the experimental treatment are administered to successive cohorts until one or more members of a cohort experience severe adverse events (SAE’s). It is at this point in the study, due to the toxic effects of the experimental treatment, that the risk of death is high. The MTD (maximum tolerated dosage) is then determined and the study stops. Again, the goal of a Phase 1 clinical trial is to determine the maximum dose of the new treatment that a human body can live through. Even though the effective dosage might be obvious, the Phase1 trial is not stopped until dosages that cause severe side effects are studied. Unfortunately, this compromises the life of some members of the study. When the Phase 1 trial is stopped, the "safest" and unsafe dosages are usually published (see an Example of a Summary Published Upon Completion of a Phase 1 Clinical Trial*) - especially if the treatment has the funding to continue on into the Phase 2 study process. Why would anyone volunteer to join a Phase 1 cancer clinical trial?The reasons are as varied as people are. Usually a desire to be of use and to help future cancer patients is high on the list of reasons to become involved in a Phase 1 cancer clinical trial. Also, there is the hope that, just maybe, this untried treatment might hold back death. In the case of a Phase 1 cancer clinical trial, that hope is minimal to none. But, the one cohort receiving the "safe" dosage may experience some benefit. Some people think that, because a Phase 1 cancer clinical trial is a scientific exercise, great care and attention will be accorded to the patient. This is not always true; actually, few to no basic human needs are supplied by the research facility to people who volunteer for a Phase 1 cancer clinical trial. The patient's medical care costs are no longer paid for by the Province. Those costs are covered by the funding supplied to the research facility by the sponsor of the study. Thus no record of medical services will appear on the patient's provincial "MediCare" file. The Phase 1 cancer study volunteer is the personification of the word "altruistic." Some “body” has to do it. Where do Phase 1 cancer clinical trials take place?Phase 1 studies usually take place in major urban centres. The clinical trial volunteer is required to re-locate to that centre for a significant period of time – particularly significant to a terminally-ill person as ‘time’ is precious. In B.C., the British Columbia Cancer Agency (BCCA) located in Vancouver is the main location conducting Phase 1 studies. Phase 2 and Phase 3 clinical trials can be conducted in smaller centres as the ‘safe’ dose of the test treatment is already established – by Phase 1 patient volunteers. As well, knowledge has been gained from the Phase 1 study on exactly what the adverse side effects are that the test treatment might induce. Hopefully, the appropriate remedy for such AE’s will have been worked out. Is a Phase 1 cancer clinical trial patient volunteer kept under constant observation during their involvement with the research study?No. You are not under constant observation - the study is performed on an Out-Patient basis; most of the time, you are on your own. You are away from home, ill and without your usual support network. Because of this, *Research with Respect* offers free support for the "away from home" time while you participate in the trial. Please see our Team Roles and Self Help Forms pages to help you ensure you receive appropriate care. How is the Phase 1 patient volunteer monitored?The patient is responsible for tracking his or her own well-being. The trial subject must understand that the researchers are interested in furthering the development and acceptance of the experimental cancer treatment that they have hitched their reputations to. The researchers, therefore, pay keen attention to the laboratory data generated by the copious samples that are extracted from the Phase 1 study subject. Phase 1 researchers follow the experimental treatment results attentively. Their focus is the experimental treatment; the patient volunteer is merely a means to an end – that end being the answer to the question, "What is the MTD (maximum tolerated dosage)?" The patient would be naive to assume that researchers attach a “face” to the lab reports that they peruse. It is the job of the research team to “keep their eyes” on the progress of the experimental treatment. The Phase 1 clinical trial patient is encouraged to contact the clinical trial nurse should the patient experience any difficulty. There is no guarantee that the trial nurse will keep a record of those calls/visits or of any advice that she might give. Thererfore, it becomes the patient’s duty to log and record this information for any future reference. In medicine – if it isn’t written down, it didn’t happen. The *Research with Respect* program helps you to help yourself by providing the following free services: • Community nurse visit If you are, or are thinking of becoming, a Phase I Cancer Trial volunteer, call Toll-Free: 1-866-265-1920 to register for this free service. (Note: The Team Roles page on this site will help you understand the roles of the people involved in a Phase I Oncology Clinical Trial. The Self Help Forms page on this site provides some handy forms and a Physical Assessment Guide you can use to keep track of your health and care. ) Normally, the Phase 1 patient would meet with the research doctor once every 2 weeks. Should the patient encounter adverse side effects, the research doctor will usually arrange to meet with the patient. Researchers prefer to not hospitalize the Phase 1 clinical trial patient as that could create the possibility that the resulting statistics may reflect badly on the experimental treatment’s future marketability. If, eventually, the experimental treatment does get to be in general use and might cause future patients to be hospitalized, then the cost of using the new treatment would soar – always a negative to budget conscious healthcare systems. Researchers prefer to attempt to remedy Phase 1 patient AE’s (adverse events) the same way that they conduct most Phase 1 clinical trials – on an out patient basis. Thus the Phase 1 patient may be directed to an Ambulatory Daycare even when a “normal” cancer patient dealing with the exact same AE /SAE’s (severe adverse events) would be hospitalized. How do I locate a Phase 1 trial?Discuss your interest in locating a Phase 1 clinical trial with your GP or oncologist. Usually, your doctors will try to dissuade you. The Phase 1 Clinical Trial Volunteer process can be a scary and lonely experience. Your own doctors will want to provide you comfort and connectedness in your end-stage of life. If you persist, efforts will be made to find a trial that addresses the particular cancer that you are dealing with. That would seem to make sense and will give you the feeling that you are still striving to beat your cancer. But when ‘push comes to shove’, the researchers are looking for a body that is just healthy enough to cope with the demands that their experimental treatment and its investigative process will make. To date, the heartening improvements in cancer care that have been achieved can be attributed, in great part, to the terminally-ill patients who have “stepped up to the plate” and joined a Phase 1 clinical trial. What can “Research with Respect” do for the Phase 1 volunteer?The real ‘doing’ is done by the Phase 1 clinical trial volunteer. “Research with Respect” assists the efforts of the Phase 1 volunteer and the volunteer’s own caregivers to obtain well-rounded medical attention. ‘Adequate’ and ‘reasonable’ are not acceptable levels of care for people who donate their lives to better the lot of their fellow human beings. ‘Optimal’ medical care is what is assured to Phase 1 clinical trial participants; that is what should be delivered. The current mind-set of researchers and medical institutions is hard-pressed to make good on its promises of optimal care. “Research with Respect” has intimate knowledge of the Phase 1 cancer research system’s weaknesses and is striving to fill the gaps in patient care. “Research with Respect” encourages and supports the heroic, terminally-ill cancer patients who bravely take on the rigors of a Phase 1 clinical trial. The goal of "Research with Respect" is to make potential volunteers aware of the role that they and their caregivers must play in the Phase 1 exercise. The volunteer must retain his or her personal power and not turn their autonomy over to the research team. "Research with Respect" provides free support, networking and tools to help in the retention of that power. How do I register for *Research with Respect*?You can register for this program yourself - we do not need your doctor to register for you, or permission from the trial researchers. All you have to do is call Toll-Free: 1-866-265-1920 to register for this free service. You will be asked for a copy of your completed 'Subject Information & Consent Form' from the study, as well as all pertinent medical information needed by the nursing staff to provide the support you need. The 'Subject Information & Consent Form' shows that you are eligible for the *Research with Respect* program. Your personal and medical information is required by Bayshore Home Health in order to provide the care you need. All information is kept confidential. | |
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