Clinical trials are conducted in all types of health diseases and disorders. You and your child may be thinking about or may have been asked to enroll in a clinical trial. Understanding what clinical trials are will help you make an informed decision about enrolling in a clinical trial. Knowing the benefits and risks of a clinical trial can also help you decide if a clinical trial is right for your family.
This section of Clinical Trials will cover:
What are Clinical Trials?
Treatment options for bleeding disorders are always changing and improving. The development and approval of new treatments relies heavily on clinical trials. These are studies that give researchers and healthcare providers important information about how well a treatment works, in what amounts and how it should be given, 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). After approval by the FDA, it can be given to the general population. Sometimes clinical trials also test different ways of diagnosing or monitoring specific health conditions.
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 moving on testing in the next one.
Clinical trials can be completed several ways. One of the most common is to use a double-blind, placebo-controlled design in phase 3 trials. 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 if a participant is receiving the experimental treatment or the existing treatment or placebo. A placebo is a treatment that’s inactive. Often called a sugar pill, a placebo 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 and your child are interested in participating in a particular trial, your child may not meet the criteria 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 your child for a clinical trial, you should discuss the trial in detail with your child’s healthcare provider to be sure you and your child understand the risk.
Here are some key points to discuss:
Treatment Reactions. Your child may experience unpleasant, serious, or even life-threatening side effects when they take the study drug.
Effectiveness. The study drug may or may not work for your child they are in the group receiving it.
Time Commitment. As part of the clinical trial, your family may need to give more time and attention to managing your child’s bleeding disorder. This may include follow-up visits, additional treatment, hospital stays, or complex dosage needs.
What Are the Benefits?
Participating in a clinical trial may be beneficial to both your family and the bleeding disorders community as a whole. When you enroll your child in a clinical trial, you may:
Play an active role in your child’s health care and the management of their 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 thought.
Here are some things to think about when enrolling your child in a clinical trial:
Are you and your child comfortable with the risks?
How will it impact your child’s everyday life?
How will it impact your family and other loved ones?
You will likely want to discuss the possibility of enrolling your child with their healthcare provider or Hemophilia Treatment Center team, as well as your family.
If you want to learn even more about clinical trials, please go to NBDF’s Clinical Trials Essentials. You can also find ongoing research studies in the Clinical Trials Database.