Clinical Trials: Information for Patients and Caregivers

Understanding what clinical trials are can help you decide if a clinical trial might be an option for you, or maybe you have a friend or family member with HAE and are wondering if a clinical trial, is right for them.

This page provides some basic information about clinical trials, to help you understand what’s involved in taking part. This includes information about the benefits and risks; who is responsible; and how your safety is protected.

What are clinical trials?

Clinical trials are research studies that involve people. They are one step in the long process that begins with research in a lab. Most treatments today are the results of past clinical trials.

HAE clinical trials are designed to test new ways to treat HAE.

Every trial has a person in charge, usually a doctor, who is the principal investigator. The principal investigator prepares a plan for the trial, called a protocol. The protocol explains, what will be done during a trial. It also contains information that helps the doctor decide if this treatment is right for you. The protocol includes information about:

  • the reason for doing the trial;
  • who can join the trial;
  • how many people are needed for the trial;
  • any drugs that will be given, how they will be given, the dose and how often;
  • what medical tests will be done and how often; and
  • what types of information will be collected about people taking part.

Why are Clinical Trials important?

Clinical trials are key to developing new treatment options for HAE. It is through clinical trials that researchers can determine wether new treatments are safe and effective and work better than current treatments.

When you take part in a clinical trial you add knowledge about HAE and help improve care.

Shire - Hamilton
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Biocryst - Edmonton (Pending Approval)
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Who is involved in Clinical trials?

Designing and running a clinical trial requires the skills of many different types of experts.

Each team may be set up differently at different sites. Typical team members and their responsibilities include:

Principal Investigator – supervises and is responsible for all aspects of a clinical trials. This person:

  • develops the concept for the trial;
  • writes the protocol;
  • submits the protocol for Ethics Board review and approval;
  • directs the recruitment of patients;
  • manages the informed consent process;
  • supervises data collection, analysis, interpretation and preparation; and
  • is responsible for the safety and welfare of each subject.

Research Nurse – manages the collection of data throughout the course of the clinical trial. This person:

  • educates staff, patients and referring health care providers about the trial;
  • communicates regularly with the principal investigator; and
  • assists the principal investigator with monitoring, quality assurance, audits and data management and analysis.

Data Manager – manages the collection of data throughout the course of the clinical trial. This person:

  • enters the data;
  • works with the principal investigator and research nurse to identify which data will be trailed;
  • provides data monitoring agencies; and
  • prepares summaries for interim and final data analysis.

Staff physician or Nurse – helps take care of patients during a clinical trial. This person:

  • treats patients according to the clinical trial protocol;
  • assesses and records how each patient responds to treatment and the side effects they may have;
  • works with the principal investigator and research nurse to report trends of how patients are doing in the treatment; and
  • manages each patient’s care.

Patient Safety in Clinical Trials

If you are thinking about taking part in a clinical trial, you may have questions such as:

  • Are there risks to taking part in this trial?
  • Who is watching out for any problems?
  • Who is making sure that participants are safe?
  • Is the trial trying to answer and important research question?

In fact, there are Health Canada rules in place to help ensure the safety and ethics of clinical trials. You will be protected through:

  • the informed consent process;
  • careful review and approval of clinical trials by Scientific Experts, Health Canada, and Ethics Review Board; and
  • careful monitoring of the trial by Ethics Board.

Paying for Clinical Trials

As you think about taking part in a clinical trial, you will face the issue of how to cover costs. Research costs are those related to taking part in the trial. Often these costs are not covered by insurance and may be covered by the trials sponsor. Expenses include:

  • the study drug;
  • lab tests performed purely for research purposes;
  • additional tests performed solely for the trial; and
  • any incidental costs to you, the patient related to participating in the study, such as parking etc.

When you take part in a trial you may have extra doctor visits that you would not have with standard treatment. These extra visits can add costs for transportation and even child care.

Informed Consent

Informed consent is a process through which you learn details about the trial before deciding whether to take part. This includes a critical part of ensuring patient safety in research.

During the informed consent process, the research team will explain the trials:

  • purpose;
  • procedures;
  • risks and benefits; and
  • chances of receiving the placebo vs study drug.

They will also explain your rights; including your right to:

  • make a decision about participating; and
  • leave the study at any time.

Before agreeing you have the right to:

  • learn about all your treatment options;
  • learn all that is involved in the trials, including all details about treatment, tests, and possible risks and benefits;
  • discuss the trial with the principal investigator, other members of the research team, your family, and/or your physician; and
  • both hear and read information in a language you can understand.

After discussing the study with you, the research team will give you an informed consent form to read. The form includes written details about information that was discussed with you and describes the privacy of your medical records. If you agree to take part in the study, you sign the form. But even after you sign the consent form, you can leave the study at any time. You can always ask questions and as new information becomes available, the research team will inform you.

Preparing for a Clinical Trial

Questions you Might Want to Ask your Doctor

  • What is the purpose of the trial?
  • Why do the researchers believe that the treatment being studied may be better than the one being used now? Why may it not be better?
  • How long will I be in the trial?
  • What kinds of tests and treatments are involved?
  • How will I be informed about the trials results?
  • How long do I have to make up my mind about joining the trial?
  • Who can I speak with about questions I have during and after the trial?
  • Who will be in charge of my care?
  • Is there someone I can talk to who has been in the trial?

Questions about Risks and Benefits

  • What are the possible side effects or risks of the new treatment?
  • What are the possible benefits?
  • How do the possible risks & benefits of this trial compare to those of the standard treatments?

Questions about your Rights

  • How will my health information be kept private?
  • What happens if I decide to leave the trial?

Questions about Costs

  • Will I have to pay for any of the treatments or tests?
  • What costs will my health insurance cover?
  • Who pays if I’m injured in the trial?
  • Who can help answer any questions from my insurance company?

Questions about Daily Life

  • How could the trial affect my daily life?
  • How often will I have to come to the hospital or clinic?
  • Will I have to travel long distances?
  • Will I have check-ups after the trial?

Questions about Comparing Choices

  • What are my other treatment choices, including standard treatments?
  • How does the treatment I would receive in this trial compare with other treatment choices?