Trials

What is a clinical trial?

Clinical trials are research studies with human participants that seek to figure out if new treatments and medications are safe, and if they work. Taking part in a clinical trial means contributing to the rapidly advancing world of medicine. Participation is always voluntary.

Why should I participate in a clinical trial?

There is no substitute for volunteers in clinical research. The knowledge we gain cannot be found in a laboratory even with our most sophisticated scientific instruments. Volunteers like you are the only way for us to advance medicine.

But why should you volunteer for a clinical study? There are many reasons:

• You may be pleased with your current treatment, but still want to assist in developing new treatments for your condition.
• You may have a loved one with an illness or injury and you want to participate as a healthy volunteer in a research study as a way of helping that person and others who may suffer from the same condition.
• Perhaps, there is no treatment for your condition that works for you, or it has harsh side effects. Participating in a clinical trial may offer potential treatment options otherwise be unavailable.
• Many people choose to participate in a study even though it might not be able to help them directly. Knowing that others may be able to benefit from their efforts can be a rewarding experience.
• Sometimes people volunteer because they are compensated for their time and effort.

Whatever the reason for volunteering, when you participate in a clinical trial, you are contributing to medical progress that benefits each of us every day. Your cholesterol medicine, your spouse’s cancer treatment, your daughter’s asthma inhaler, the latest Alzheimer’s drug that your dad is taking – all of these are available to you because of clinical studies that show they are safe and effective. None of them would be possible without volunteers who were willing to take part in clinical studies.

Will I be compensated for clinical trials?

Many of our trials provide compensation for time and travel. Compensation should never be the primary reason to volunteer, but we value your contribution to our research and are happy to discuss these details when you are considering a study.

What is a placebo drug?

Placebos are harmless substances that are designed to look like the active medicine used in the clinical trial. Placebos allow the researchers to objectively evaluate how well the study drug works when compared to those not receiving it.  Many “placebo-controlled” studies are followed by a period called “open-label” where all participants receive the active medicine. You will always know before enrolling if your study has a placebo and what the chances are of you receiving it. For example, the study may be designed where 20% of participants receive placebo. In this scenario, 4 out of 5 people receive active medicine.

What is a randomized study?

This is a study in which participants are divided by chance into separate groups that compare different treatments or placebo. By using chance to divide people into groups, these groups will be similar so that the effects of the treatments they receive can be compared more fairly.

What are single-blind and double-blind clinical trials?

In both forms of study, the participants do not know what medicine is being used. The purpose of blinded studies is to prevent bias from patients and staff. In a single-blind study, only the patient is not told what is being given, while the staff are aware. In a double-blind study, the pharmacist preparing and administering an intervention is the only person who knows which treatment (or placebo) is being given. The ultimate goal of clinical research is to collect scientifically accurate data; performing single-blind and double-blind studies is the best way to achieve this.

What are the risks involved in participating in clinical trials?

As with any activity, there is some risk involved in clinical research. However, it is important to consider the degree of harm a clinical trial might cause. To best understand risks associated with a study, participants must consult the protocol for that study specifically, because risks vary from study to study. The research team, led by a principal investigator, will ensure that all participants are aware of any risks prior to beginning.

What is the definition of a healthy volunteer?

A healthy volunteer is somebody with no known significant health problems who participates in research to test a new drug, device, or intervention. There are two types of participants in a study: healthy volunteers and patient volunteers. These volunteers are recruited to serve as controls for patient groups, and they play a key role for a study in the collection of data.

The study I am considering is “Phase III”. What does this mean?

Human clinical trials are divided into three main phases and can be followed by post-marketing surveillance studies known as phase IV.

1. 1. Phase I clinical trials are the first step in testing new drugs and treatments on humans. The aims of these smaller-scale trials are to assess the safety of experimental drugs, to observe any possible side effects, to determine safe dosages, and to see how the body reacts to the treatment.
   2. Phase II clinical trials occur after the initial safety of the treatment is demonstrated in Phase I. They are designed to analyze the therapeutic efficacy of a treatment in people who have the condition being studied. Phase II trials can be divided into two phases, Phase IIA and Phase IIB. Phase IIA is designed to assess dosages for a drug, and to look for side effects in a larger patient pool. Phase IIB studies focus on the efficacy of a treatment, such as what types of a specific disease would be best for this treatment.
   3. Phase III trials are the final stage before clinical approval of a treatment. These trials seek to demonstrate the efficacy and safety of the treatment, to monitor side effects, to test different doses if necessary, to determine if the drug or treatment could be used through the different stages of a disease, and to provide enough information for marketing approval. Phase III trials might also be used to test the efficacy of a treatment for diseases other than the one being studied.
   4. Phase IV, or post-marketing surveillance studies, seek to study the long-term effects of a treatment once it is on the market. Pharmaceutical companies that sponsor Phase IV studies describe these trials as pharmacovigilance, or the assessment of medical drugs after they are licensed for use.