In recent years, there have been several significant advancements in the diagnosis and treatment of hemophilia and other bleeding disorders.
These include extended half-life products that enable people with hemophilia to do well with fewer infusions; monoclonal antibodies that allow people to manage their hemophilia with subcutaneous injections instead of IV infusions; new assays to improve the accuracy of von Willebrand disease diagnosis; and gene therapy for hemophilia, which can potentially increase a person’s factor levels to normal or close to normal.
None of these medical breakthroughs would have been possible if it weren’t for people with bleeding disorders deciding to participate in a clinical trial. Read on to learn about how clinical trials work, and how to find the information you need about clinical trials for hemophilia and other bleeding disorders.
How Clinical Trials Work
A clinical trial is a research study that gives scientists and health care providers important information about how well a novel treatment works, in what amounts and how it should be given, and/or in which patients it should be used.
Before any drug, device, or treatment is approved by the Food and Drug Administration (FDA), it must first be tested in a clinical trial. Most clinical trials are interventional studies, which are designed to compare new medical, surgical, or behavioral approaches with existing treatment options.
Clinical trials have four phases:
- Phase 1 is focused on safety and involves a small group of healthy volunteers.
- Phase 2 is focused on safety and effectiveness and involves a small group of people with a target disease or condition.
- Phase 3 is focused on safety and effectiveness and involves a larger, more diverse group of people with a target disease or condition.
- Phase 4 is focused on specific populations or safety monitoring. This phase occurs after a treatment has been approved by the FDA.
Phase 3 of clinical trials, which is the last step before a new treatment is approved, often use a double-blind, placebo-controlled design. This means that some participants receive the drug that is being studied, and some receive either standard treatment or a placebo. It’s called double-blind because neither the participants nor the health care providers know if someone is receiving the experimental treatment or the existing treatment or placebo.
To be eligible to participate in a clinical trial, a person must satisfy both inclusion criteria and exclusion criteria. Inclusion criteria define who may enroll in a trial, while exclusion criteria define who may not enroll. Exclusion criteria may include age, gender, diagnosis, and coexisting health conditions.
How to Find Clinical Trials for Hemophilia and Other Bleeding Disorders
The U.S. government’s National Library of Medicine maintains ClinicalTrials.gov, an online database of clinical research studies from around the world.
People with hemophilia or other bleeding disorders can search the database by entering their specific condition or disease. The database allows you to narrow your search by entering eligibility criteria, interventions or treatments, and locations. You can select
“Recruiting and not yet recruiting studies” to narrow your search to studies that are looking for participants.
The health care team at your hemophilia treatment center is another great resource for information about participating in clinical trials. They can answer your questions about existing trials and help you determine if a particular study is the right fit for you.
Learn about what you need to know to be a part of the latest research.