What is a clinical trial?
What is the benefit for clinical trial participants?
Is there a clinical trial for PNH?
Are there other studies in progress to help understand PNH?
A clinical trial is a scientific study of potential new treatments or medications for a particular disease or condition. It is used to evaluate possible treatments that have potential effects against the disease/condition. Scientists may test whether a treatment is simply effective and safe, or if one treatment is more effective than another. Of course, how well a drug is tolerated is also a focus. A clinical trial involving human subjects is part of the long and careful process in the evaluation of a treatment candidate. If a new treatment or medication is proven effective AND safe, it may become approved for use in a specific population.
What is the benefit for clinical trial participants?
By participating in a clinical trial, a person helps to provide valuable information about his or her disease and possible treatment options. Participants may find the research to be beneficial to themselves, though this is not always the case. A trial may use a placebo (an inactive substance that helps determine a potential treatment’s safety and effectiveness when compared to an active drug or treatment) for some of those involved, or the therapy in question might work for only certain participants and not others. There is also a chance that a patient might experience an adverse event or side effect from the use of the drug. Further information can be found in the resources section of PNHSource, including additional background on clinical studies.
Is there a clinical trial for PNH?
There have been several clinical trials for PNH, but enrollment
is now closed.
Visit the National Institutes of Health or ClinicalTrials.gov for
more information.
Are there other studies in progress to help understand PNH?
Yes! One study that is currently taking place is called the Enhanced Examination of PNH with Laboratory Observations and Research in Bone Marrow Disorders, or simply EXPLORE. The EXPLORE study is designed to examine the frequency of PNH in patients with aplastic anemia (AA), myelodysplastic syndrome (MDS), and other bone marrow failure disorders.
Also, your physician can enroll you in the PNH Patient Registry—a multinational observational research initiative designed to help improve patient understanding of and treatment options for
the disease.
Only a physician can enroll a patient in a study or clinical trial. Make sure to talk to your physician if you are interested in participating. To learn more about available options, click here.
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