What is a clinical trial?
A clinical trial is considered a biomedical or health-related research study. They may be referred to as a study, a trial or a protocol. There are two designations for these studies; interventional and observational.
Interventional studies are those in which the participants (research subjects) are assigned to a specific treatment or intervention by an investigator/doctor. Participants are carefully monitored and the outcomes are measured.
Observational studies are just that. Participants are observed and their outcomes are measured by an investigator.
The individual leading the clinical trial is called the Principal Investigator, and this person is usually a specialist in the area being studied. There will be several Associate Investigators who also take part in leading and organizing the clinical trial. In addition to the investigators, you may meet other members of the healthcare team including nurses, pharmacists and social workers.
Clinical trials are divided into three main phases, Phase I, Phase II, Phase III and Phase IV, according to the purpose of the trial. When new drugs are being investigated they must first be tested in a Phase I trial. A Phase I trial will determine the safety of the drug in humans and may determine maximum tolerated doses of the drug. Phase I studies are not done to determine a drug's effectiveness although observations may be made about participants' conditions if they are affected. Phase II studies are designed to use the information learned in the Phase I trials to plan interventions that may produce a desired effect in a participant. If there appears to be a significant benefit to the participants, a Phase III trial may be planned. Phase III trials expand these interventions to a larger number of people allowing the investigators to make assessments of efficacy, side effects, as well as its safety in a larger population. Phase IV trials are large studies of individuals taking a drug or using a new treatment after it has been approved and is on the market. These studies are important to the health of the public as new drugs and treatments are more widely used.
The most important aspect of a clinical trial is the Informed Consent. Informed Consent is both a process and a document. Informed Consent should be an ongoing communication between the investigators and the trial participants. As new information is learned or as changes are made, the participants should be informed. This communication should take place throughout the protocol.
The Informed Consent is also a document prepared to inform the participants about the clinical trial, what the trial's goals are, how long it lasts, any procedures, tests or treatments the participant may undergo and any risks or benefits to participation in the study. You should be provided your own copy of the Informed Consent Document. If the document is written in English and your native language is different, please ask for the document to be translated for you.
Informed Consent documents are signed and dated by both the Investigator and the Participant. Informed Consents ARE NOT CONTRACTS. The participant is not obligated to continue participating in the trial and may decide to stop participating at any time.
Why participate in a trial?
Being part of a clinical trial is just one way of taking a more active role in your own health and CML journey. Through a trial, not only can you gain access to therapies that might to be widely available or currently on the market. Additionally, you are contributing to medical research and allowing the investigators to gain greater insight in the treatment of your disease.
How do I get involved in a trial?
Each trial has certain guidelines that must be followed. Certain criteria is used to ensure that the research done will provide reliable results, thus participants must meet certain inclusion or exclusion criteria. These criteria may include factors such as age, gender, disease stage, previous treatment history, or other medical conditions. It is important to realize that these criteria are not used to include or exclude you PERSONALLY, they are used to identify appropriate candidates and to ensure the safety of those participating.
How does a trial work/what happens?
First off, you will be assigned a trial team, which may include your present doctor. Others involved are nurses, social workers, and other healthcare professionals. The team will evaluate your current state of health and give specific instructions on participation. Additonally, they will monitor you closely during the trial and stay in touch with you after the trial is completed.
Some trials involve more tests and doctor visits than you might normally experience when seeking treatment for CML. it is important that you understand that strict adherence to the trial protocol is not only important for the research team and study, it is important for your health and safety.
What is Informed Consent?
Prior to your agreeing to take part in a clinical trial, you will be informed of all details so that you can make the decision that is right for you. The information will give you key facts about the trial, including details of the study, its purpose and duration, required procedures, and key contacts. You will also be asked to sign an Informed Consent Document that outlines the potential risks and benefits of the study. Although you must sign this document to take part in the trial, the document is NOT a contract and you may withdraw from the study at any time.
What are some risks and benefits of participating?
As with anything in life, there are risks and benefits. Carefully considering these items are important to insure that you are fully informed about what will take place in the trial.
Benefits - clinical trials that are well-designed and executed are the best approach for participants to:
- take an active role in their own health care.
- gain access to new treatments before they are widely available to the general public.
- obtain expert medica care at leading health care facilities
- help others by contributing to medical research
Risks - although every precaution in take, there are risks associated with participating in a clinical trial.
- possible serious or even life-threatening side-effects to experimental treatment
- unpleasant procedures
- the treatment may not be effective for you
- the protocol may require more time and attention that non-protocol treatment, including more treatments, trips to a study site, hospital stays, or complex dosage requirements.
What questions should I ask when considering a clinical trial?
You should know as much as possible about any trial you are considering. Ask members of your care team questions about the trial, what type of care can you expect during the trial, and the costs involved. Here are some questions you might ask:
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the experimental treatment being tested may be effective and has it been tested before?
- What kinds of tests and experimental treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Who will pay for the experimental treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the experimental treatment is working? Will results of the trial be provided to me?
- Who will be in charge of my care?
I've heard of a study that combines Gleevec and Interferon to achieve a cure. What is the theory and is it sound?
There are currently trials for combination therapy, the most popular being the Gleevec®/Interferon trial. Dr. Moshe Talpaz at the University of Michigan has been conducting this study, as well as other institutions.
There have been a small number of patients that were initially treated with Interferon and then later with Gleevec® that have been able to stop therapy for an extended amount of time. It is not yet fully understood what allows some to stop therapy without their CML returning, therefore it is unwise for any patient to arbitrarily stop therapy if they have had Interferon and Gleevec®. You should discuss ANY change of dosing/frequency with your treating physician.
For more information, visit http://www.nationalcmlsociety.org/living-cml/clinical-trials and use the search links to location a center conducting this combo trial.