About Clinical Studies

Deciding to participate in a clinical trial is an important personal decision. This section should provide you with detailed information about clinical trials. You can always contact our study research team to ask questions about specific trials. It is also helpful to talk to a physician, family members, or friends when deciding to join a trial.

What is a clinical trial?

Typically, medical research studies involving people are called clinical trials. Clinical research is one of the ways the medical world looks for better or new ways of treating illness and disease. The U.S. Food and Drug Administration (FDA) requires that all medications be thoroughly tested for safety and effectiveness before they can be marketed in pharmacies and prescribed by your doctor.

The Department of Health and Human Services (DHHS) defines a clinical trial as a controlled study involving human subjects that is designed to prospectively evaluate the safety and effectiveness of new drugs, devices, or of behavioral interventions.

What are the benefits for participants?

Qualified participants in clinical trials:

Can play a more active role in their own healthcare by helping with the evaluation of new medications or devices.
May get new research treatments that could work for the treatment of your condition that could not be obtained outside the trial.
Help the future of others, and yourself, by participating in medical research.
Receive closer monitoring and associated medical care. All visits and procedures related to the study are free of charge. Those may include physical examinations, doctor visits, EKGs, dietary counseling, study medications, and laboratory tests.
May receive compensation for participation in the trial.

Who can participate?

People from all over the world participate in clinical trials in order to help themselves and others improve their quality of life. Volunteer participants must qualify for the clinical trial. Clinical trials have guidelines about who may qualify because each clinical trial attempts to answer specific research questions. These guidelines are called inclusion/exclusion criteria.

Inclusion criteria are those factors that allow someone to participate in the clinical trial.

Exclusion criteria are those factors that disallow someone from participating.

These inclusion/exclusion criteria are important in medical research and help to produce reliable results, identify appropriate participants, and keep them safe. These criteria are based on factors such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.

What happens during a clinical trial?

All volunteer patients are treated professionally and with care. Participating in a clinical trial is much like a regular visit to a doctor’s office, hospital, or medical clinic. Our clinical trial team always includes doctors and nurses as well as other healthcare professionals. At the beginning of the trial, they check the health of the participant and give specific instructions for participating in the trial. During the clinical trial study, medication is administered. The participant is monitored carefully during the trial and tests and procedures are performed according to a research protocol. This research protocol is designed to measure the effectiveness and safety of the study medication or procedures.

What are the risks for participants?

There will be some risk and participants are always closely monitored. Treatment will be stopped if the study doctor thinks the risks outweigh the benefit for you.

The risks may include:

• The investigational treatment may not be effective for some participants.
• There may be unpleasant, serious, or even life-threatening side effects from the trial treatment.
• The protocol may require more of the participant’s time and attention than the participant can give.

Even in phase 1 or 2 trials, the treatment product has been carefully researched in the laboratory before it is administered to patients.

What are the side effects and adverse reactions?

Side effects are any adverse events or effects of the investigational drug or treatment. Such effects may include such events as headaches, nausea, hair loss, skin irritation, or other physical problems.

You will be informed of everything that the doctors know regarding the study product before you agree to take part in the trial. However, in phase 1 of trials, the researchers may not have sufficient information to advise you of side effects. It is important to note that you may withdraw from a trial at any point. Your participation is entirely voluntary.

What are the phases of a clinical trial?

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions. There are four different phases of clinical trials. These phases are called phase 1, 2, 3, and 4:

Phase 1: Phase 1 trials are the earliest trials in the life of a new drug or treatment. The investigational drug is tested in a small group of people (20-80) to establish safe dosage ranges, identify side effects, learn how the body copes with the drug, and determine if the treatment actually treats the disease. The participants entering phase 1 are often healthy individuals. Phase 1 trials are important because they are the first step in finding new treatments for the future.
Phase 2: Phase 2 trials are conducted with a larger group of people (100-300). Phase 2 determines if the new treatment works well enough to test in phase 3. It tests the effectiveness and safety and identifies side effects.
Phase 3: Phase 3 trials are conducted with a much larger group of people (1,000-3,000) to confirm product effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the investigational drug or treatment to be used safely.
Phase 4: Phase 4 trials delineate additional information including the drug’s risks, benefits, and optimal use, also addressed are post-marketing safety studies.

What is a protocol?

A protocol is the study plan for all clinical trials. This plan safeguards the health of the participants as well as answer specific research questions. The 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.

What is a placebo?

A placebo is an inactive substance with no treatment value. Investigational treatments are often compared with placebos to assess the investigational treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or investigational treatment.

What is a control group?

In many clinical trials, one group of participants may be given the investigational drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

What if I already have a doctor?

You should continue to see your doctor for all non-study-related medical matters and discuss your participation in the clinical trial.

Do I get paid to participate in a clinical trial?

You may receive compensation for time and travel while you are participating in the study. Volunteers are not charged for study visits and procedures.

The preceding information is sourced from:

Your Safety and Protection

Are Clinical Trials Safe?

Strict guidelines are followed and experts review the studies to ensure patients are never subjected to undue risk. However, studies may involve some degree of risk. Participant safety is the number one priority in any study.

Each participant is given a consent form to sign which describes the most common risks for the medication being studied, the potential benefits of participating in the study, and expectations of persons participating. Participants will be able to discuss complete informed consent with the research clinic staff. They will help you fully understand the risks and potential benefits of participating in the study and your rights as a research participant. Your medical condition will be monitored throughout the study by experienced medical staff and physicians.

How is the Safety of the Participant Protected?

The ethical and legal codes that govern medical practice also apply to clinical trials. All clinical research is federally regulated with built-in safeguards to protect the participants. Treatments have been thoroughly tested in laboratory trials before they are ever tested with groups of patients. Medications must be FDA-approved in phase 1 and 2 trials before they can be used in phase 3 and 4 trials.

There are many other safeguards for patients taking part in trials including the following:

The trial protocol is a study plan that details what researchers will do in the study. It is reviewed and must be approved by an external ethics review committee (Internal Review Board/IRB) before implementation.
All aspects of the clinical trial are supervised for the duration of the trial. A data monitoring committee and researchers review and report research findings during the course of the trial.
Participant privacy is protected by HIPAA (Health Insurance Portability and Accountability Act of 1966), so individual participants’ names remain secret and are not mentioned in any reports.
Participants are fully informed of the benefits and risks before they volunteer to take part.

What is Informed Consent?

The informed consent form includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are also explained in this document. The participant then decides whether or not to participate and sign the document. The informed consent form must be signed by participants; prior to the initiation of any study procedures. This is not a binding contract and the participant may withdraw from the trial at any time.

This process allows the participant to gain information about the clinical trial before deciding to volunteer to participate. Patients must be provided with all known details about the trial when considering their consent to participate.

What is the Role of the FDA?

The Food and Drug Administration (FDA) ensures that medical treatments are safe and effective for people to use. The FDA requires that all medications are thoroughly tested for safety and effectiveness before they may be prescribed by a doctor. The FDA does not develop new therapies, or conduct clinical trials. It functions strictly as a regulatory body. The FDA meets with researchers and performs inspections of clinical trial study sites to protect the rights of participants and to verify the quality and integrity of the data collected during a study.

What is Good Clinical Practice?

Good Clinical Practice (GCP) guidelines are a standard which has been created for the design, conduct, monitoring, auditing, recording, analysis of results, and reporting of clinical trials. Both the FDA and IRB adhere to GCP guidelines. These guidelines are applied in clinical research trials in order to provide assurance that the final results which are reported are accurate and credible and that confidentiality and rights of trial participants are protected.

What is the Role of the IRB?

An Institutional Review Board (IRB), also known as an Independent Ethics Committee (IEC) or Ethical Review Board (ERB), is a committee that has been formally designated to approve, monitor, and review biomedical and behavioral research involving humans. The purpose of these boards is to protect the rights and welfare of the research subjects. In the United States, the Food and Drug Administration (FDA) and the Department of Health and Human Services (specifically Office for Human Research Protections) have regulations that empower IRBs to approve, require modifications in planned research prior to approval, or disapprove research. An IRB performs critical oversight functions for research conducted on human subjects that are scientific, ethical, and regulatory. They ensure that participants involved in research projects are protected from undue risk. They also ensure that subjects voluntarily give informed consent and that their safety, rights, privileges, and privacy are protected.

What is the Common Rule?

According to the U.S. Department of Health and Human Services, the Common Rule is a rule of ethics regarding biomedical and behavioral research involving human subjects in the United States. These regulations governing Institutional Review Boards for oversight of human research came into effect in 1981 following the 1975 revision of the Declaration of Helsinki, and are encapsulated in the 1991 revision to the U.S. Department of Health and Human Services Title 45 CFR 46 (Public Welfare) Subparts A, B, C, and D. Subpart A ("The Common Rule") is the baseline standard of ethics to which any government-funded research in the U.S. is held. However, all academic institutions and researchers are held to these statements of rights regardless of funding sources.

What are the Alternatives to Participating in a Clinical Trial?

It is important to consider alternatives to clinical trials, such as treatment that is already deemed safe and approved by the FDA, or even treatments that do not involve medications.

Can I Leave or Drop Out of the Clinical Trial After it has Begun?

Yes. All qualified participants may leave a clinical trial at any time. You should discuss your situation with the research team regarding your decision and the reasons for leaving the study.

Preparing for your Study Visit

How to Prepare:

Participants should feel comfortable asking the members of our research team any questions about the clinical study. It is also important for you to know as much as possible about the clinical study. The Informed Consent Process will help you learn all the key facts about the study. You may also review the sections on 'About Clinical Studies' and 'Your Safety and Protection' (found above).

General Guidelines:

Participants will be given specific instructions prior to each study visit relative to the protocol. In general, participants may be asked to do one or all of the following:

Follow the directions of your study coordinator regarding the study medication prior to your visit.
If fasting: Do not eat food for a specific period of time prior to your visit.
If fasting: Continue to drink water during the fasting period prior to your visit.
Return study-related diaries, the investigational drug, logs, forms, or special equipment to the visit.
Advise the clinical research coordinator of any change in medications you may be taking.
Inform the clinical research coordinator of any illnesses or related events since your last visit.
Expect that the average visit will involve 30-90 minutes of your time.

Do I Continue to Work with my Primary Healthcare Provider While in a Study?

Yes - continue to follow the treatment regimen prescribed by your primary care physician. Your healthcare provider may even work with our research study team and ensure that other medications or treatments will not conflict with the protocol. The study does not take the place of your personal healthcare agenda.

Important Clinical Study Questions to Ask and Consider

What is the purpose of the study?
Who is going to be in the study?
Why do researchers believe the investigational treatment being investigated may be effective?
Has the investigational treatment been tested before and what phase is the study in?
What kinds of tests and treatment choices are involved in this trial?
What are the advantages and risks of participating?
What are the possible side effects?
How do the possible risks, side effects, and benefits of the study product compare with my current treatment?
How long will this trial last?
How many times will I have to come to the clinic?
Will I have to be hospitalized?
How will I know if it works? Will I be told about the results?
How might this trial affect my daily life?
Who will be in charge of my care?
Will my primary care physician know about this trial?
Can I leave the trial if I want to?
Who can I contact in an emergency?
Who makes sure the trial is safe and properly run?
Am I covered by insurance?
Do I need medical insurance to participate in the trial?
Will I be compensated for my participation? What about any expenses that I might incur, such as traveling costs?
Who will be allowed to see my medical records?