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The Leukemia/Bone Marrow Transplant Program of BC

General Information
Research
Frequently Asked Questions - FAQs

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Click on any of the questions below to skip to the answer.

  1. What is clinical research?
  2. What is a clinical trial?
  3. What are the phases of a clinical trial and what do they mean?
  4. How are study patients protected?
  5. How is my confidentiality protected in a study?
  6. What are my rights if I participate in a trial?
  7. Am I eligible?
  8. What questions should I ask the study staff?
  9. What questions to ask yourself to decide if entering a clinical trial is right for you?
  10. Where can I find more information?

1. What is clinical research?

Research is an incremental process that moves forward in small and carefully planned steps. Usually research begins as basic science in the lab and after years of testing in cells and tissues, testing is done on animal models of human cells. After treatments prove successful in animals, these treatments may be tested on people through clinical trials.

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2. What is a clinical trial?

A clinical trial is a research study designed to answer questions about safety and effectiveness of a drug or treatment. These trials follow a rigorous scientific process with built-in safeguards for participants who volunteer and are selected for research. Most clinical research involves the testing of a new drug in an orderly series of steps called phases. The process starts with small trials testing the safety of a treatment and then moves towards progressively larger trials. The larger trials compare the safety and effectiveness of the new therapy to the currently accepted standard of care.

It is important to remember that in a clinical trial, researchers do not know if the new treatment will be more effective than the current standard of care treatment.

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3. What are the phases of a clinical trial and what do they mean?

Note that to participate in a clinical trial, patients must first meet screening criteria.

PHASE I TRIALS
Summary
  • Earliest trials in the life of a new drug or treatment
  • Usually smaller trials; recruitment is low from 15 to 50 patients
  • Since Phase I trials are usually the first time a drug is given to a patient, the first few patients, or cohorts, are given a small dose of the drug.
  • The dose is then gradually increased with each group. The effect is monitored until the best dose is determined.
  • Phase I trials require significant time commitment from participants because they involve frequent blood tests and side effects monitoring.
  • Often patients with a cancer that lacks or does not respond to standard treatment are participants in Phase I trials.
Objectives
  • To determine safe dose range
  • To determine side effects
  • To determine how the drug works in the body (pharmacologic behaviour) and how the body copes with and excretes the drug (pharmacokinetic studies)
  • To determine how well the drug works (efficacy)
Benefits
  • If the new drug or therapy has an effect on the cancer, participants may be the first to benefit
Risks
  • Unpredictable side effects can occur

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PHASE II TRIALS
Summary
  • To evaluate the effectiveness in a larger population, usually less than 100 patients
  • Often focus on cancers for which no effective treatment exists or that are most likely to show a response to therapy
  • All participants receive the same dose
Objectives
  • To assess effectiveness of drug or therapy
  • To assess for additional safety information
Benefits
  • May benefit if treatment has an effect on the cancer
Risks
  • Unpredictable side effects may occur
  • Trials are often too short to determine long term benefits

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PHASE III TRIALS
Summary
  • Large trials with 100+ participants
  • Multi-centre, often global
  • Survival time and quality of life measured
  • Subjects are ‘randomized’ to a group: the Study Group — receiving study therapy, or the Control Group — receiving standard of care
Objectives
  • To determine whether a new therapy or drug is more effective or less debilitating than standard of care
Benefits
  • May be among the first to benefit if drug or treatment is shown to work
Risks
  • New treatments are not always better than or even as good as standard of care
  • Side effects may be worse than standard of care
  • Unexpected side effects may occur despite preceding phase I and II trials
  • Subjects in the Control Group who are receiving standard of care may receive less benefits than those in the Study Group who are receiving the study regimen.

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4. How are study patients protected?

A committee of nurses and doctors at the Leukemia/BMT Program reviews all clinical trials proposed by the Leukemia/BMT Program. The purpose of the Review Committee is to ensure that the trials are clinically and scientifically sound.

Once the protocol has been accepted by the Leukemia/BMT Program Review Committee, the study protocol must be approved by an Ethics Board. Before a trial can go ahead, the Ethics Board must be satisfied that the trial addresses an important scientific question, is well planned, is as safe as possible for the patients, and is ethically sound.

Before participating in any clinical trial, patients must sign a consent to ensure that they are fully informed and thoroughly understand the study protocol and the risks and benefits of their participation.

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5. How is my confidentiality protected in a study?

Your medical information is confidential, but individuals involved in monitoring the conduct of the study and recording information on the treatments may need to access your records in the presence of a study investigator. These individuals may be representatives of the study sponsors, such as the National Cancer Institute of Canada (NCIC) or the drug company supplying the trial drug. They may also be representatives from regulatory bodies such as Health Canada's Health Protection Branch.

Any forms or paperwork recording your information and results will identify you by code number only, and your name will never appear in any publication resulting from the study.

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6. What are my rights if I participate in a trial?

You do not waive, or sign away, your legal rights by signing consent to a study. Taking part in a study is voluntary. This means you may choose not to take part, or may withdraw your consent and leave the study at any time. If you withdraw from or decline a study, your doctor will discuss further treatments with you and continue to treat your cancer with the best means available. If there are other clinical trial opportunities relevant to you later in your treatment, these will be offered to you.

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7. Am I eligible?

In addition to you deciding whether the trial is right for you, your hematologist will have to make sure you are right for the trial. All participants must meet the eligibility criteria as written in the study protocol. After the consent form is signed, tests to determine the status of your disease and other baseline tests, such as blood, urine, or heart tests may be necessary to determine your eligibility.

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8. What questions should I ask the study staff?

  • What treatment options are available to me?
  • Why is this study being done? What other studies with this medication have been done?
  • Will I get a placebo?
  • What inconveniences might I face, as part of participating in the trial?
  • How much additional time is required to participate in this trial? How long is the study?
  • What are the main side effects of the study medication? What are the possible risks to me, compared to standard treatment?
  • What are the pros and cons of participating in this clinical trial?
  • Will there be reasonable reimbursement of the cost of my travel for study visits?
  • What if I change my mind? What happens when the study is over?
  • Can I take my regular medications while participating on the study?

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9. What questions to ask yourself to decide if entering a clinical trial is right for you?

  • Do I feel comfortable receiving a treatment whose risks and benefits are somewhat unknown?
  • Can I dedicate the time needed to participate in this trial?
  • Will I be able to manage if taking part in the trial means I might miss more work?
  • Am I satisfied that my decision is right for me, and not a decision that is being made to please someone else?
  • Do I have supportive family, friends or caregivers who can help me if I experience special problems such as nausea, tiredness, headaches, diarrhea?

10. Where can I find more information?

Click here for a list of additional web links.

 

The information in this section is cited with permission from Dana-Farber Cancer Institute and the BC Cancer Agency web sites.

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