What are clinical trials?
Clinical trials are medical research studies in humans. These are ways to help researchers and health care professionals find ways to improve health care. Research studies try to find new and improved approaches to diagnosing, treatment, monitoring, or preventing diseases.
The majority of clinical trials study whether a new medicine, at times in combination, is safe and effective for the treatment of a specific medical condition. These can be new medications, or an already existing medication but for a new indication. In the clinical trials the effectiveness of the study medicine is compared to a standard accepted treatment, or against a placebo if no standard treatment exists.
What types of clinical trials are there?
There are several types of clinical trials which are called “phases.” Each phase represents a more advanced stage in the development of a new medication
- Phase I: Testing a new medicine in a small group of healthy people, or people with specific conditions or diseases. The safety of a new medicine, its potential dose, and side effects are evaluated. During Phase I the researchers and the subjects (patients) are aware of the medicine which is being used.
- Phase II: The medicine is tested on a larger group of people who have a medical condition, usually to determine the efficacy of the medication. Like Phase I, the patients and researchers are aware of the treatment.
- Phase III: This is a large study; usually it is conducted in many centers and in several countries. The medicine is compared to acceptable treatment or to placebo. Phase III trials are used to apply for FDA approval. Phase III trials are designed as randomized, double-blind studies; neither the participants, nor the doctors or the researchers, know which person is getting the study medication or the standard treatment.
- Phase IV: Once a medication has been approved, it may be studied for different indication or treatment combinations. This phase is also usually designed as randomized double-blind study or open-label.
What is the role of clinical trials?
Clinical trials may find treatment and cure to medical conditions. They help companies develop medicines that are safer and more effective than existing therapy. They are especially important in finding treatments for conditions that have no available treatment. Clinical trials help identify how new treatments work and what are the possible side effects.
What in it for me?
Participating in a study will make you part of a select group which defines the future of science and medicine. When in a study you will benefit from comprehensive medical evaluation and care, and you will be closely supervised and adequately educated to help you achieve a good control of your medical condition. Your evaluation is free of charge, as well as the study medications. At times all your medications for the particular medical condition of the study may be covered as well. Many studies provide a modest stipend to cover expense and effort.
How does it work?
We will determine whether you are eligible to participate in a clinical trial based on a set of criteria that you must meet. The study will be explained to you in detail. Once you agree to participate, you will be asked to sign a consent form.
Provided you meet the criteria of the study based on evaluation and laboratory tests, it will be decided to which arm (part) of the study you belong. You will be assigned, usually by a computer, to one of the treatment arms. Based on which type (phase) study it is, you and the researcher may not know which treatment you are getting.
You will then be given a program to follow. You will have to take medications, see the doctor periodically, undergo further tests, and may be asked to keep a diary of your progress and adverse effect. At the end of the trial you will return all study medications.
You will not be able to continue with unapproved study medications but for special circumstances – e.g. compassionate treatment if it was helpful for a severe disease, or if you are offered by the sponsors of the trial to continue the study.
Once I come for screening, do I have to participate?
Participation in a clinical trial is voluntary. You will be informed of the study procedures and the possible risks and benefits of the trial. You will be asked to agree to be part of the study and to sign a consent form to participate.
You may choose not to participate in the study and ask to receive standard treatment for your condition – at which time you may be referred back to your health care provider. At other times we may be able to accommodate your needs. Occasionally, participation in the trial may not benefit you directly; however it may help other people who have the same condition as you.
Are there risks associated with the trial?
All medications and treatment modalities carry some risks. You will be fully informed about the possible risks and benefits of the trial before you consent to participate, so you would know what to expect. The higher the phase of the study, we typically anticipate less serious side effects.
Some of the risks associated: the new treatment may not work as well as standard treatments. Side effects, especially in early phases, may be significant. You should notify your doctor/researcher of any unusual symptoms. You will be very closely monitored and cared for should any adverse effect occur.
What am I sacrificing?
You are not sacrificing much, as the reward and the satisfaction are often high. You should be prepared to: spend more of your time than usual for treatment; take more medications; come more often to the research site; be subjected to multiple tests; keep complex diaries and treatment regimens. However, the more complex the study is, more will be required from you, along with a higher stipend to be offered to you. And in the end you may benefit from your experience.
How is my safety protected?
Clinical trials are usually designed under the FDA guidelines and approval. Studies need to also be approved and monitored by a special body: the Institutional Review Board (IRB). The IRB makes sure that the study is ethical, that the risks are as low as possible, and that potential benefits outweigh the risks. Clinical trials typically have data safety monitor boards to further protect the safety of patients. Many time the results of the study are published, but your name and identity are not revealed.
How can I participate in a clinical trial in your center?
You can call the Metabolic Center at 818 342 1889; or Dr Yehuda Handelsman’s office at 818 708 9942; and leave a message for Mike Heinzel or Dr. Handelsman. You can call Mike directly at 818 681 3750.