Clinical Trials 101 | Crohn’s & Colitis Foundation

Clinical Trials 101

Understanding the Basics of CCFA Studies and Clinical Trials

What is a clinical trial?

Clinical trials are simply biomedical or health-related research studies in patients that follow a pre-defined protocol (or set of rules to follow). Outcomes of these trials are measured by the researchers or investigators who initiated the study.

Why should you participate?

By participating in a medical study or clinical trial, you can have a more active role in your own health care, gain access to new research treatments before they become widely available, and help others by contributing to medical research.

How long can a clinical trial take (from start to drug approval)?

The process of drug approval can be a lengthy one. According to the Tufts Center for the Study of Drug Development at Tufts University, it takes an average of 15 years for an experimental drug to go from lab to patient. Your participation in a trial can last over a year (though you may withdraw at any time) and it may take even longer to analyze the results.

What happens during a clinical trial?

Researchers begin by testing compounds (drug preparations involving several ingredients), adding them to enzymes, cell cultures, or cellular substances to see which combinations improve the compound's performance. If a compound succeeds in the test tube, it's time to test it on animals. Researchers generally test drugs on two or more species, because the agent might affect each differently. These tests determine any toxic side effects and its safety at various doses. Animal testing also helps researchers to see how a drug is absorbed into the bloodstream, and how quickly it is excreted.

Phase I (Human Testing - primarily for safety): Once the drug is successful in animal testing, clinical trials begin in a small group of patients, typically healthy males, to demonstrate:

  • Safety
  • Appropriate dose range
  • How the drug is metabolized (broken down and cleared by your body) in order to determine the best way to administer treatment

According to Tufts, 5 in 5,000 compounds that undergo preclinical testing make it to human testing (and one of those five is approved).

Phase II (Pilot Trials to establish what is the best dose): In this phase, a slightly larger group of patients will be tested to determine:

  • If the treatment works
  • What are the best doses to test in a larger population.

Phase III (Larger Trials): If the pilot trials are promising, then the Phase III trials demonstrate:

  • Safety and effectiveness in larger trials involving hundreds of patients.

In the U.S., if two large Phase III trials are positive, the drug can be presented to the FDA in a New Drug Application (NDA). If approved, the drug can then be marketed for the specific indication, such as to maintain remission in ulcerative colitis. However, once approved, doctors can prescribe for any indication.

Phase IV: In some studies, a Phase IV is necessary. In this phase, the researchers are sometimes required to extend the prior indication at different doses, dose schedules, or formulations.

What types of protocols do clinical investigators need to follow?

The rules and regulations of a clinical trial are designed by the sponsor. This is usually a pharmaceutical company, although research institutions and health organizations also fund investigations. The sponsor designs a protocol—a detailed description of how the trial should be conducted—for clinical investigators, the physicians who conduct the trial from various locations.

The protocol will describe the type of trial involved. Often, preliminary trials are open label, meaning that patients receive the experimental medication at a dose that is known to the patient and the doctor. In controlled trials, some participants receive the trial drug while others receive a placebo (a harmless substance that looks identical to the drug on trial).

Many trials are randomized, meaning patients are randomly assigned to receive either drug or placebo. Studies also can be double-blinded, meaning that neither patient nor physician knows whether drug or placebo is being taken, or at what dosage. “Blinding is important to prevent bias in the results.  Sometimes if a patient or physician knows drug is being given, depending on their feelings about the drug, they may subjectively think things are improving or worsening.  If they don’t know the treatment being given, then any response to treatment reported is more likely to be accurate.

According to FDA regulations, an institutional review board, (IRB) made up of healthcare professionals and lay people from the facility and community where the trial is taking place, keeps tabs on that entity's involvement in the trial. The IRB makes sure that all FDA and protocol regulations are adhered to, and reviews patient recruitment, advertising, and potential risks.

What should you ask if you are thinking about participating?

Enrolling in a trial is a careful process for the patient as well. CCFA plays a part in bringing trials to the attention of potential participants. However, the patient should discuss participation with his or her personal physician, and that physician should be kept posted about the patient's progress in the trial.

Patients enrolling in a trial are closely monitored throughout by the clinical investigators. On the first visit, the patient should be ready with a list of questions and concerns:

  • What is the purpose of the trial and how long will it last?
  • Who is the sponsor?
  • Who has reviewed and approved the trial?
  • What kinds of tests and treatments are involved?
  • Will there be any pain or discomfort?
  • What are my treatment options, other than this medication?
  • What are the advantages and disadvantages?
  • How often will I be examined?
  • What side effects may occur?
  • Will the treatment be free?
  • If I am harmed, what treatment would I be entitled to?

The FDA protects patient rights by demanding that people enrolled in trials sign an informed consent form. This requirement mandates that the researcher provides adequate information about the study and responds fully to participants' questions. The investigators must be certain that the patient understands all risks and responsibilities and is aware of other treatment options.

For further information, call Crohn's & Colitis Foundation's IBD Help Center: 888.MY.GUT.PAIN (888.694.8872).

The Crohn's & Colitis Foundation provides information for educational purposes only. We encourage you to review this educational material with your health care professional. The Foundation does not provide medical or other health care opinions or services. The inclusion of another organization's resources or referral to another organization does not represent an endorsement of a particular individual, group, company or product.

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