What are clinical trials?

A “clinical trial” (or “clinical research”) is a research study involving patients and volunteers enabling us to answer specific questions related to healthcare. Carefully conducted clinical trials are the quickest and safest way to discover new and effective treatments and therapies.

For instance “interventional trials” determine whether experimental treatments are safe and effective under controlled environments. “Observational trials” however address health issues in large groups of people or populations in natural settings.

How can a patient or volunteer benefit by participating in a clinical trial?

Participants who are involved in clinical trials can play a more active role in their own healthcare and gain access to new research treatments before they are widely available. Our volunteers and patients help others by contributing to medical research.

Who is eligible to participate in a clinical trial?

All clinical trials have guidelines specifying who is eligible to participate. ‘Inclusion’ and ‘exclusion’ criteria are an important principle in medical research that enables us to produce reliable results. The factors that determine eligibility are called “inclusion criteria,” whereas the factors that determine non-eligibility are referred to as “exclusion criteria.” The decision is based on factors such as age, gender, one’s type and stage of a disease, previous treatment history and other medical conditions.

Before joining a clinical trial a participant must qualify for the study. Some research studies seek participants with specific illnesses or conditions, while others simply need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead the criteria are used to identify appropriate participants that will enable safe and accurate research results.

What events take place during a clinical trial?

The clinical trial process depends on the type of research being conducted. The clinical trial team includes doctors, nurses, social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating during the trial, monitor the participant carefully throughout and stay in touch after completion. Some clinical trials involve more thorough tests and doctor’s visits than the participant would normally have for an illness or condition. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.

What is meant by “informed consent?”

“Informed consent” is the preliminary process in which a volunteer or patient learns about a clinical trial before deciding whether or not to participate. It is also an ongoing process throughout the study to provide regular information and feedback to the participants. Upon our first consultation, doctors and nurses involved in the trial explain the details of study helping the participant to decide whether or not to engage. Translation assistance can be provided for any non-English speaking patients.

The research team then provides an “informed consent” document that includes details about the study such as its purpose, duration, required procedures, key contacts, statements of risks and benefits. The participant then decides whether or not to sign. It is important to note that the document it is not a formal contract and therefore the participant may withdraw from the trial at any time.

What are the benefits and risks of participating in a clinical trial?

It’s true that there are always risks involved in the development of ground-breaking therapies in medicine. Therefore we always have a responsibility to make the patient aware of all possible scenarios and situations. Good research design attempts to maximize benefits and minimize risks.  By carefully designing, executing and supervising trials, researchers try to reduce those risks. Participants van benefit by:

  • Playing an active role in our own healthcare.
  • Gaining access to new research treatments before they are widely available.
  • Obtaining expert medical care at leading health care facilities during the trial.
  • Contributing to medical research.

The potential risks include:

  • Ineffective treatment
  • Extended time and attention including more trips to the study site
  • More treatments, hospital stays or complex dosage requirements
  • Possible unpleasant, serious or even life-threatening side effects

Our team will educate the participant on all of the potential benefits and risks involved, ensuring safety and reliability throughout the entire process.

What are some possible side effects that a patient might experience?

Our team takes every precaution to avoid any procedure that is deemed to be unsafe for a particular patient. Side effects of experimental drugs or treatments can range from very minor to severe. For instance negative effects might include headache, nausea, hair loss, skin irritation or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects. Through consistent dialogue with the patient and ongoing evaluation, it is our responsibility to make the participant aware of every possible outcome.

How do we ensure the safety of our participants?

All 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 the participants. The trial follows a carefully controlled protocol: a plan that outlines what researchers will do throughout 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 names of individual participants will remain confidential and will not be disclosed in these reports.

What are some important questions a participant should ask in preparation for a trial?

Participants must learn as much as possible about the clinical trial prior to commencement. They should feel comfortable asking members of the healthcare team any questions that come to mind. As a start the following questions might be helpful for the participant to consider. Some of the answers to these questions are found in the informed consent document.

  • What is the purpose of the study?
  • Who is going to be participating in the study?
  • Why do researchers believe that the experimental treatment being tested might be effective?
  • Has my particular treatment been tested before?
  • What tests and experimental treatments are involved?
  • How do the possible risks, side effects and benefits of the study compare to those prevalent in my current treatment?
  • How might this trial impact my daily life?
  • How long will the trial last?
  • Will I be required to be hospitalized?
  • Who is responsible to cover the financial cost of the experimental treatment?
  • What expenses are subject to reimbursement?
  • What type of long-term follow-up care is required for this study?
  • How will I know that the experimental treatment is working?
  • Will results of the trials be provided to me?
  • Who will be taking responsibility for of my care?

In preparation for an initial meeting with the research coordinator and doctor, participants should:

  •  Plan ahead and write down possible questions to ask.
  • Ask a friend or relative to come along for support and hear the responses to each question.
  • Bring a tape recorder to record the discussion.

Every clinical trial in Canada must be approved and monitored by an Institutional Review Board (IRB) to make sure the potential benefits outweigh the anticipated risk. An IRB is an independent committee of physicians, statisticians, community advocates and other healthcare professionals ensuring that a clinical trial is ethical and protects the safety of the participant. All institutions that conduct or support biomedical research involving people must by federal regulation have an IRB approval and be subject to regular reviews.

Does a participant continue to work with a primary healthcare provider while participating in a trial?

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary healthcare. By having the healthcare provider work with the research team the participant can ensure that other medications or treatments will not conflict with the protocol.

Can a participant leave a clinical trial after it has begun?

Yes. A participant can leave a clinical trial at any time. When withdrawing from the trial the participant should inform the research team and convey reasons for departure.

Where do the ideas for trials come from?

Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial more and more information is gained about an experimental treatment, its risks and its effectiveness.

Who sponsors the clinical trials?

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, pharmaceutical companies and federal agencies. Trials can take place in a variety of locations such as hospitals, universities, doctors’ offices or community clinics.

What is referred to as a ‘protocol’?

A protocol is a document that describes the objective(s), design and the procedures involved in a clinical trial. The protocol usually also gives the background and rationale for the trial. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

What is referred to as a ‘placebo’?

A placebo is an inactive pill, liquid or powder that has no treatment value. It is a simulated treatment for a disease or other medical condition. Sometimes patients given a placebo treatment will have a perceived or actual improvement in a medical condition, a phenomenon commonly called the “Placebo Effect.” In medical research placebos are given as acontrol treatment that is directly compared to the experimental treatment as a way to assess the effectiveness of the study.

What is referred to as a ‘control’ or “control group?”

Experimental observations are evaluated in relation to a standard or a ‘control’. In many clinical trials one group of patients will be given an experimental drug or treatment, while the ‘control’ group is given either a standard treatment or a placebo. By comparing the results from each group the research team will draw possible conclusions.

What are some of the various types of clinical trials?

There is a variety of clinical trials performed today that enable us to effectively treat conditions and prevent disease. Here are some examples of the studies we conduct:

  •  Treatment trials test experimental therapies, new combinations of drugs, or new approaches to surgery or radiation therapy.
  •  Prevention trials look for better ways to prevent disease. During these procedures participants might require medicine, vitamins, vaccines, minerals or lifestyle changes.
  •  Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
  •  Screening trials test the best way to detect certain diseases or health conditions.
  •  Quality of Life trials (or Supportive Care trials) explore new ways to improve comfort and the quality of life for individuals with a chronic illness.

What is meant by ‘Phase I’ through ‘Phase IV’ in reference to a clinical trial?

Clinical trials are conducted in Phases I through IV. The trials in each phase have a specific purpose and help scientists answer different questions.

Phase I trials: Researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range and identify any side effects.

Phase II trials: The experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

Phase III trials: The experimental study drug or treatment is given to large groups of people (1,000-3,000) to:

  • Confirm its effectiveness
  • Monitor any side effects
  • Compare it to commonly used treatments
  • Collect information that will allow the experimental drug or treatment to be used safely

Phase IV trials: Post marketing studies delineate additional information including the risks, benefits and optimal use of any particular drug.

What is an “expanded access” protocol?

Most human use of investigational drugs takes place during controlled clinical trials in order to assess their safety and efficacy. Data from the trials can serve as the basis for the drug marketing application. However, sometimes patients do not qualify for these carefully controlled trials because of other health problems, age or other factors.

For patients who may still benefit from the drug but don’t qualify for the trials, FDA regulations enable manufacturers of investigational drugs to provide “expanded access” use.

The primary intent of a protocol is to provide access to the new drug for people with a life-threatening or serious disease for which there is no good alternative treatment. A secondary purpose of a protocol is to generate additional information about the drug, especially regarding its safety.

Expanded access protocols can be undertaken only if clinical investigators are actively studying the experimental treatment in well-controlled studies or if all studies have been completed. There must be evidence that the drug may be an effective treatment in patients such as those to be treated under the protocol. The drug must not expose patients to unreasonable risks given the severity of their disease.

This FAQ page is provided by CanadaTrials.com and contains content from the U.S. National Library of Medicine (NLM) and ClinicalTrials.gov.