Clinical trials are studies of drugs that are still in development or have not yet been approved to treat patients with the specific condition being studied. A drug being studied in a clinical trial is an investigational drug. These drugs are in the process of being tested for safety and effectiveness during clinical trials.
Clinical trials give information about whether the drug has intended effects, what side effects are associated with the bug, how a drug is broken down by the body, and which drugs and substances can be used with the medication.
By participating in clinical trials, patients help advance what we know about new drugs. In doing so, clinical trial participants can play an important role in getting new drugs approved for use in treating other patients. Participants in a clinical trial can also play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research. (For instance, the Liver Institute of Virginia offices in Newport News and Richmond, Virginia is currently holding a new clinical trial to test an investigational drug called obeticholic acid (OCA). Researchers hope that this man-made drug, which simulates bile acids in the liver, may be able to help patients who are diagnosed with primary biliary cirrhosis (PBC). The Liver Institute is currently recruiting interested PBC patients, who are interested in gaining access to the latest available drugs and treatments for their liver disease.)
Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:
- Phase I Trials – Researchers test an experimental drug or treatment in a small group of people (20-30) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
- Phase II Trials – The experimental study drug or treatment is given to a larger group of people (100-300) to see if its 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 side effects, compare it to a commonly used treatment, and collect information that will allow the experimental drug or treatment to be used safely. Fewer patients may be required when evaluating an investigational drug for relatively rare disease.
- Phase IV Trials – Post marketing studies delineate additional information including the drug’s risks, benefits, and optimal use.
Patients should know as much as possible about the clinical trial and feel comfortable asking the members of their healthcare team questions. For instance, “What is the purpose of the study? Has it been tested before? How might this trial affect my daily life? Who will pay for the experimental treatment? How will I know that the experimental treatment is working?” are all good questions.
If you are considering participating in a clinical trial, like those at our Liver Institute of Virginia, you should carefully read information about the drug trial you are considering and ask questions about exactly what will happen during the trial . You are entitled to received detailed information regarding what is known about the drug before you decide to enter a clinical trial, and you must provide a signed informed consent document.
Source: Intercept Pharmaceuticals information.