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Clinical Trials

Treatment options for bleeding disorders are constantly evolving and improving. The development and approval of new treatments relies heavily on clinical trials—studies that provide researchers and health care providers with essential information about how well a treatment works in a controlled situation, how it should be dosed or administered, and/or in which patients it should be used. Every drug, device, and treatment must be tested in a clinical trial before it is approved by the Food and Drug Administration (FDA) and used in the general population. Sometimes clinical trials also evaluate different methods of diagnosing or monitoring specific health conditions.

Clinical trials are conducted in all types of health diseases and disorders. As a person with a bleeding disorder, you may be considering or may have been asked to enroll in a clinical trial. Understanding what clinical trials are—and are not—and knowing the benefits and risks involved can help you make an informed decision about whether enrolling is right for you.

Types of Clinical Trials

There are 4 types of clinical trials, identified as phase 1 to phase 4. A treatment must be proven successful in one phase before progressing to testing in the next one.

Phase 1 Trial

This is the first time a study drug is tested in humans. These trials involve a small number of people and help researchers evaluate drug safety, determine a safe dosage range, and identify side effects.

Phase 2 Trial

The study drug is usually given to a larger group of people than in phase 1 trials to determine drug effectiveness and compile more information on drug safety.

Phase 3 Trial

The study drug is given to an even larger number of people to confirm drug effectiveness, safety, and how it compares with other already-licensed drugs.

Phase 4 Trial

These trials occur after a drug has been approved by the FDA to provide continued safety information and efficacy information.

Clinical trials can be conducted a number of ways. One of the most common in phase 3 trials is to use a double-blind, placebo-controlled design. In this type of trial, some participants receive the study drug and some receive standard treatment or a placebo. In double-blind trials, neither the participants nor the health care providers know whether a participant is receiving the experimental treatment or the existing treatment or placebo. A placebo is a treatment that’s inactive; it’s often called a sugar pill, although it can be a liquid or even an injection.

Every clinical trial is designed with inclusion criteria and exclusion criteria.

  • Inclusion criteria define who may enroll in the trial
  • Exclusion criteria define who may not enroll in the trial. These criteria often include age, gender, diagnosis, and other health conditions a person may have

You should know that even if you are interested in participating in a particular trial, you may not be eligible (meet the criteria required) for enrollment.

Are Clinical Trials Safe?

All clinical trials are designed to be as safe as possible for the participants. Some risk is typical when not much is known about a study drug. If you’re thinking about signing up for a clinical trial, you should discuss the trial in detail with your health care provider to be sure you understand the risk.

  • Here are some key points to discuss:

    • Treatment Reactions. You may experience unpleasant, serious, or even life-threatening side effects when you take the study drug
    • Effectiveness. The study drug may or may not be effective (work for you)—even if you’re in the group receiving it
    • Time Commitment. As part of the clinical trial you may need to devote more time and attention to managing your bleeding disorder with follow-up visits, additional treatment, hospital stays, or complex dosage requirements

What Are the Benefits?

Participating in a clinical trial may be beneficial to both you and the bleeding disorders community as a whole. When you enroll in a clinical trial, you may:

  • Play an active role in your own health care and the management of your bleeding disorder
  • Gain access to new research treatments before they are widely available
  • Obtain expert medical care at leading health facilities during the trial
  • Help others with bleeding disorders by contributing to medical research

Making the Decision

Enrolling in a clinical trial requires careful consideration.

Here are some considerations when enrolling in a clinical trial:

  • Are you comfortable with the risks?
  • How will it impact your everyday life?
  • How will it impact your family and other loved ones?

You will likely want to discuss the possibility of enrolling with your health care provider or Hemophilia Treatment Center team, as well as your family.

To learn more about clinical trials, go to Clinical Trials.