What is a clinical trial?

A clinical trial is a research study of how a drug, medical device, or treatment approach works in people. Each study answers scientific questions and tries to find better ways to prevent, screen for, diagnose, or treat a disease. Clinical trials may also compare a new treatment to a treatment that is already available.

Doctors and other health professionals run the study. They follow strict rules to test how well a new drug or a new medical approach works. When you volunteer to be in a study, you might try a new treatment or drug that may or may not be better than one that is already in use. The doctors and researchers don't know ahead of time how the study will turn out. That is why they are doing the research.

In the U.S., there are strict rules to make sure that people who agree to be in a clinical trial are treated as safely as possible. Every research study has an action plan, or protocol, that describes what will be done in the study and how it will be carried out. This plan is reviewed and approved by a committee at each place doing the clinical trial. This review helps make sure that the risks of the study are small and are worth the possible benefits.

Why participate in a clinical trial?

Being part of a research study can mean playing a more active role in your health care. It can also:

  • Help you gain access to new treatments before they are widely available to the public
  • Offer expert care at leading health care facilities during the trial
  • Help others by contributing to medical research

Being in a clinical trial is not for everyone. The information in this section will help you learn more about research studies before you decide if this option is right for you.

Why are clinical trials important for rare diseases?

  • Treatment options are often limited for rare diseases. These studies help doctors find new and better ways to find, prevent, diagnose, treat, and control rare health problems.
  • Research studies help doctors find out the best dose and schedule to use when giving a treatment. This improves a treatment's success rate. Studies are also a great way to find better treatments with fewer side effects.
  • Past research studies have led to the approval of many effective treatments for bone marrow failure diseases. These include:
  • decitabine (Dacogen)
  • deferasirox (Exjade)
  • lenalidomide (Revlimid)
  • eculizumab (Soliris)
  • azacitidine (Vidaza)

Research has also helped develop new procedures, such as bone marrow transplants. Clinical trials explore and measure ways to improve the comfort and quality of life of people with a chronic illness through supportive care.

What are some risks of being in a clinical trial?

Before you join a clinical research study, you should know that:

  • The treatment being tested may not work as well as the standard treatment. The term "standard treatment" means a treatment that has been tested and found helpful and that is commonly used.
  • There is a chance that the side effects of the treatment being tested may be more serious than those of the standard treatment. The risks depend on the type of treatment you receive. The study researchers will explain all known risks to you.
  • You may have more doctor visits and tests than if you were not in the study.

What happens in a clinical trial?

These studies test whether new products are safe and work against a disease. A research team may include doctors, social workers, and other health care professionals. They will check your health at the start of the study and give you instructions for being in the trial. They will check you carefully during the entire study and may stay in touch with you when the study is done.

Some studies require that neither you nor your doctor know whether you are getting the new study treatment, the standard treatment, or a placebo. Clinical trials may compare a new medical approach to a standard one that is already available or to a placebo that contains no active ingredients or to no intervention. Some clinical trials compare interventions that are already available to each other.

What is a clinical trial protocol?

Before a research study begins, the lead researcher creates a study plan called a "clinical trial protocol." The plan lists exactly what will happen during the study and why.

Most protocols include:

  • The specific research questions the study hopes to answer
  • Who may take part in the study
  • How many participants are needed
  • How long the study will last
  • The results the doctors hope to see in response to the treatment
  • What information will be gathered about the participants

Protocols also address the medicine and treatment and include:

  • The medicine to be used and the dosage
  • How the medicine or treatment will be given
  • Any lab tests that will be done
  • Known side effects of the treatment
  • Possible benefits and risks of the treatment

The protocol must be reviewed and approved by a special review committee, called an Institutional Review Board, or IRB for short. Doctors, scientists, and people from the community make up an IRB. These IRB members review the study plan to make sure that the rights of volunteers in the study are protected. They also check to see that the risks of being in the study are reasonable in comparison to the expected good outcomes.

Who can be part of a clinical trial?

Some research studies look for people with certain health problems to be studied. Other studies need only healthy participants. Every clinical trial has a set of guidelines about who can be part of that study. These guidelines are also called eligibility requirements. The factors that allow someone to participate in a clinical study are called inclusion criteria. Those factors that disqualify someone from participating are called exclusion criteria. These are based on things such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.So, before joining a clinical trial, you must qualify for the study.

Before you are allowed to be in a study, you'll be asked to fill out a form to see if you meet the guidelines. The guidelines help the researchers make sure people with the health condition being studied are in the trial. These guidelines also protect people who have certain health problems that may make it dangerous for them to be part of the research study.

Often, the guidelines are set so that people in the study are as much alike as possible. This way, the doctors and researchers can be sure that the results from the study are due to the treatment.

You may be asked if you:

  • Have a certain type or stage of illness
  • Have had a certain kind of therapy in the past
  • Are in a certain age group