Frequently Asked Questions for Cancer Trial Volunteers

How are new cancer treatments developed?

New cancer treatments, like treatments for other diseases, are created by combining new concepts and compounds with scientific knowledge gathered from past bio-medical research efforts. The resulting new cancer-fighting possibilities are then developed in laboratories and tested on animals. The next step is to use the experimental treatment on live human beings. In order for a new pharmaceutical product to receive marketing approval from the responsible government authority — in this country, that is Health Canada’s (HC) Therapeutic Products Division — it must be tried-out on people. Clinical research trials are the testing of investigational drugs or medical devices on live human beings. For a new cancer drug, the clinical trial process can take 10-12 years to complete.

How is a cancer clinical research trial structured?

In a clinical trial, three separate phases of inquiry are carried out.

  • Phase 1 testing determines the dosage of the investigational drug that is safe for use on humans.
  • Phase 2 testing determines if that ‘safe’ dose is effective.
  • Phase 3 testing determines if the safe dose of the new drug is better, at least in some ways, than those currently in use.

Approximately 10% of clinical trials produce a product that receives marketing approval. 90% of all investigational drugs fail at some point in the clinical trial process for various reasons – the drug doesn’t work, the side effects are horrendous, the sponsoring company runs out of money, etc.

Focus on Phase 1

The Phase 1 Clinical Trial – the first time an investigational medical product is used on human beings – is pivotal to the entire marketing approval process.

  • A Phase 1 Clinical Drug Trial is often referred to as a ‘safety study’ because discovering the safe dose is the point of the exercise. However, ‘toxicity trial’ more accurately describes how that safe dose is arrived at.
  • Most Phase 1 investigational cancer drug trials are ‘escalating dose’ studies. The dosage amount that a Phase 1 volunteer starts with is the dose they will continue to receive throughout their association with the trial.
  • The first small group of Phase 1 volunteers is administered a dosage that the lab animals were able to tolerate. If no side effects or only manageable ones (AEs) occur, then another group of 2-4 people – a cohort – is recruited and the dose amount is increased. This ‘recruit a new cohort / increase the dose’ pattern continues until a cohort experiences severe adverse effects / events (SAEs) – physical symptoms that could cause death.
  • The schedule for increasing the dosage is set by the sponsor of the trial – usually a pharmaceutical company that has developed the drug. That dosing schedule is vetted by Health Canada and by the research ethics board of the institution that has a contract with the sponsor to conduct the trial. In Canada, 80% of all investigational drug trials are funded by pharmaceutical companies.
  • When a toxic or lethal level or dose-limiting toxicity (DLT) --severe side effects--are experienced by members of a cohort, the Phase 1 trial is stopped and deemed to be a success. It is then possible for researchers to back-track through the accumulated data to determine the Maximum Tolerated Dose (MTD) and then pin-point the safe dose.
  • Typically, a Phase 1 clinical trial requires 20-40 volunteer subjects being exposed to the investigational drug over a period of 18 months.

Again, the goal of a Phase 1 clinical trial is to determine the maximum dose of the new treatment that a human body can stand. Even though the ‘safe’ dosage might become obvious, the Phase 1 trial is not stopped until dosages that cause severe side effects are experienced and recorded.

When the Phase 1 trial is stopped, the safe and unsafe dosages are usually published (see an example of a Phase 1 clinical trial summary *) - especially if the treatment has the funding to continue on into the Phase 2 clinical trial process.

Phase 2: Once the safe dosage is determined by the Phase 1 study, the experimental treatment is tested in a Phase 2 study to see if it is an effective anti-cancer therapy. For a Phase 2 trial, only patients with the particular cancer that the investigational drug was designed to combat are recruited. A Phase 2 clinical trial usually requires between 100 and 300 subjects.

Phase 3: If the new treatment is found by the Phase 2 study to be useful in treating cancer, then it is compared, in a Phase 3 study, to therapies currently in use. Placebos might be incorporated into a Phase 3 trial. A Phase 3 clinical trial can involve a few hundred to a few thousand test subjects.

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Who are the Phase 1 cancer clinical trial subjects?

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 people, all previous possible avenues for cure or containment of their cancer have been exhausted. Not all persons terminally ill with cancer can be accepted into a Phase 1 study. The prospective Phase 1 subject’s body must meet a specified level of adequate major organ function before entering the study. In a Phase 1 trial, it doesn’t matter what kind of cancer the person is dying of, just that they possesses a human body still capable of processing the investigational drug. If their body passes the rigorous pre-enrolment tests, the volunteer must agree to provide numerous blood samples for laboratory analysis throughout their entire association with the Phase 1 trial and, further along in the trial, agree to have more detailed testing – CAT scan, etc.

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Do Phase 1 cancer trial volunteers receive compensation?

Phase 1 cancer trials are usually conducted on an “out-patient” basis. The participants don`t receive any financial compensation and must pay all costs incurred for transportation, accommodation, food, over-the-counter medications, parking, etc.

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Why do people volunteer to join a Phase 1 cancer clinical trial?

The reasons are as varied as people are. Many people who volunteer for Phase 1 trials express the wish to “do something positive with their life,” to be of use, to contribute to science and to help future cancer patients. Some people volunteer because they hope that this untried treatment might have a therapeutic benefit and halt or slow down the progress of their disease. In the case of a Phase 1 cancer clinical trial, there is almost no chance that the treatment will have any positive effect. At best, there is a slight possibility – less than 4% - that the one cohort receiving the "safe" dosage may experience some benefit. If you are considering volunteering for a phase 1 cancer clinical trial, it is important to remember that the purpose of this trial is just to determine the maximum tolerated dose or toxicity level of the drug, not to provide any personal health benefit. What will my care be like if I join a Phase 1 cancer trial? If you decide to participate in a Phase 1 clinical trial, you will work with a research team. Team members may include doctors, nurses, and other health care professionals. They will provide your care and give you specific instructions about the study. The Team Roles page will help you understand the roles of the people involved in a Phase I cancer trial. You will typically be treated on an out-patient basis. Accommodation, transportation and any support care you need will be your own responsibility. In many cases, few to no basic human needs are supplied by the research facility to people who volunteer for a Phase 1 cancer clinical trial.

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What will my care be like if I join a Phase 1 Cancer Trial?

If you decide to participate in a Phase 1 clinical trial, you will work with a research team. Team members may include doctors, nurses, and other health care professionals. They will provide your care and give you specific instructions about the study.  The Team Roles page will help you understand the roles of the people involved in a Phase I cancer trial.

You will typically be treated on an out-patient basis. Accommodation, transportation and any support care you need will be your own responsibility. In many cases, few to no basic human needs are supplied by the research facility to people who volunteer for a Phase 1 Cancer Cinical Trial.

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Where do phase 1 cancer clinical trials take place?

Currently, Phase 1 cancer studies are conducted in only ten major cities across Canada. The clinical trial volunteer is required to temporarily take up residence in rented accommodations as near to that research centre as possible for a significant period of time—typically 30 days. Many blood samples are required at very specific times, especially throughout the first week after the experimental drug has been administered to the volunteer. In order to maintain the integrity of the data collected, Phase 1 volunteers must punctually present themselves at the research centre’s laboratory. It is for this reason that you must relocate to the near vicinity of the research centre to be close to the lab facilities that are tracking the test drug. This lengthy re-location requirement can have a significant impact as the comforts of home and community are stripped away at this pivotal time in your journey. Phase 2 and Phase 3 clinical trials can be contracted out to smaller centres as the ‘safe’ dose of the test treatment is already established – by the Phase 1 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.

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How is the phase 1 cancer trial volunteer monitored?

Normally, the Phase 1 volunteer would meet with the research doctor once every 2 weeks during the study. Should you encounter adverse side effects, the research doctor will usually arrange to meet with you. You also may be encouraged to contact the clinical trial nurse should you 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. Therefore, you should keep your own record of this information for any future reference. (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.)

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What is the difference between my own physician and the research doctor?

The important difference between your own physician and the research doctor is their goals. The number one goal and responsibility of your physician is to prescribe the best known course of treatment for you and to make recommendations promoting your health and well-being. The goal of the research doctor (and research team) is to recruit volunteers and to maintain the integrity of the research data collected so as to achieve a successful completion of the portion of the Phase 1 trial that they have been contracted to conduct. Thus, your care and well-being is not the primary objective of researchers and health professionals involved in the trial.

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What is a Principal Investigator?

Health Canada requires there to be a health care professional, usually a physician, who is in charge of a clinical trial at each institution that has a portion of the Phase 1 Clinical Trial contract. This individual is referred to as the Principal Investigator (PI) at that site. There is always a Principal Investigator for the overall clinical trial as well, but that individual may not be based at the particular site to which you are reporting.

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How do I find a Phase 1 clinical trial?

Discuss your interest in locating a Phase 1 Clinical Trial with your family doctor or oncologist. Your doctor may try to dissuade you as volunteering for the Phase 1 Clinical Trial process can be a scary, lonely and even dangerous experience and your own doctors will want to provide you comfort and connectedness in your end-stage of life. If, however, you are determined to volunteer, you can ask your family doctor or oncologist if they know of any trial that is recruiting subjects.

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How are participants in phase 1 trials protected?

The physical safety of the participants of Phase 1 Clinical Trials is completely dependent upon the Phase 1 research team's conscientiously adhering to the plan laid down for conducting the trial. This ‘plan of action’ is called the protocol. The protocol is developed by the sponsor of the trial with input, vetting and final approval being provided by the contracted institution’s Research  Ethics Board (REB) and by Health Canada. However, on a day-to-day basis during the ‘away-from-home’ period, it is the responsibility of the Phase 1 trial participant to be vigilant and meticulous in reporting any changes in their level of well-being to the Phase 1 team. It is therefore advisable to enlist the attentions of a knowledgeable third party whose focus is solely on the welfare of the Phase 1 volunteer. Such an independent third party could be the local hospice society, the palliative care team attached to the local health authority or the Research with Respect Care Program provided by the ‘Bear Ring "U" 'n Mind’ Foundation. Of course, once the Phase 1 volunteer returns home, their personal health care team’s expertise can then be easily accessed.

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What is a Clinical Trial Protocol?

Before a government-funded or privately-funded clinical trial can begin, the trial plan (also called a protocol) must be approved. The trial protocol – proposed by the sponsor of the trial - must be approved by the contracted institution’s Research Ethics Board and further vetted by Health Canada. Health Canada and the Research Ethics Board require researchers conducting clinical trials to thoroughly inform prospective volunteers about a trial’s treatments and tests and the possible benefits and risks before that person decides whether or not to participate in the clinical trial. This process is called “informed consent.”

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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, they will be included in a written consent form that you can take home to read and discuss with others. Once you qualify to be accepted into the trial, you must sign the ‘Subject Information & Consent’ form. A physician, usually exclusively contracted to the institution which has been contracted by the sponsor, will sign and a witness – possibly your personal caregiver - must sign as well. 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.

• Your responsibilities.

• The contracted institution’s responsibilities.

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 trial, 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. For more information, see Questions You Should Ask and Clinical Drug Trial Consent Form information.

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