Researchers have made dramatic progress in diagnosing and treating pulmonary hypertension in all its forms. Ongoing research continues to provide fresh insights into the processes that lead to pulmonary hypertension and new ways to treat it.

But much work remains to be done. Many drugs are effective for only a small proportion of patients. The disease still poses a life-threatening danger for many people. For that reason, a wide variety of clinical trials are under way or actively recruiting volunteers -- investigations designed to learn more about the disease process and to test promising therapies. Researchers are conducting clinical trials not only for pulmonary hypertension but for the many disease conditions that can lead to it, from autoimmune disorders to chronic lung conditions. Several are looking at pulmonary hypertension in people with sickle cell disease, for instance. Others are studying genetic factors in primary pulmonary hypertension.

Clinical trials are the means by which new drugs and treatments are tested to determine whether they work. They are the engine that drives progress in medicine. In addition, for patients who have run out of other options, clinical trials offer a last chance at a potentially effective therapy. In some cases they provide access to research treatments before they are made widely available.

It's important to remember that clinical trials often involve unproven therapies. There is no guarantee that the treatments under investigation will work. And there is always a chance that they may cause serious side effects. Still, by participating in clinical trials, patients help advance the science of medicine.

If you or someone you know is interested in participating in a clinical trial, there are some important things to remember.

Clinical trials have specific guidelines about participation. Not everyone can qualify to be part of every clinical trial. To make sure clinical trials are safe and provide reliable scientific information, researchers carefully choose who can -- and who can't -- take part. Clinical trials have "inclusion criteria" (the requirements for participating) and "exclusion criteria" (factors that disallow people from taking part). These are usually spelled out in descriptions of clinical trials made available to the public.

Participants must be informed and give their consent. Doctors, nurses, and other health professionals involved in running a study are required to explain the details of the investigation to prospective participants. They provide an "informed consent" document with the details of the study explained in writing. Participants are asked to sign the document. Additional information is sometimes provided during the study, as it becomes available.

Participants are always free to withdraw from a study. Even after signing an informed consent document and joining a clinical trial, volunteers can withdraw at any time. Withdrawing can jeopardize research, however, so it's important to learn everything you can before agreeing to join a trial.

Not everyone in a study always receives the active drug or treatment. Some studies test an active drug against a placebo. A placebo is an inert substance such as a sugar pill: It's used in clinical trials to compare the effect of a given treatment against no treatment. In blinded studies, neither participants nor researchers are allowed to know who is getting the real drug and who is being given the placebo. (A sealed code is used to keep track for later analysis.) Blinded studies are essential to eliminate bias in interpreting the results.

Typically, if a drug appears to be working effectively, researchers will decide to "unblind" the study and offer the new treatment to everyone in the study. Not all studies involve a placebo, however. Some test the safety of a new drug. Others are designed to better understand the process of a disease.

Clinical studies are conducted in four phases. Phase I trials test a new drug or treatment in a small group of people to make sure it's safe, to determine a safe dosage range, and to look at side effects. Phase II trials usually include more people and study whether the drug is safe and whether it's effective.

Phase III trials involve still more participants. These tests are designed to confirm a treatment's effectiveness, monitor its side effects, and sometimes compare it to commonly used treatments. In Phase IV trials, drugs that have already been approved are studied for more information about their benefits, uses, or potential risks.

Investigational drugs are sometimes offered outside of clinical trials. The FDA allows manufacturers of investigational drugs to offer them to people who might benefit but who don't qualify for clinical trials. This "expanded access," as it's called, is usually offered to people with a serious or life-threatening illness. The drug must already have been shown to be safe and effective in well-controlled studies.

For more information about clinical trials, talk to your doctor or check out these comprehensive sites:

National Institutes of Health

http://www.ClinicalTrials.gov

This site provides detailed information about clinical trials and has a search feature that locates ongoing clinical trials.

CenterWatch

http://www.CenterWatch.com

This site includes a listing of clinical trials organized by state.

-- Peter Jaret is a contributing editor for Health magazine and a winner of the American Medical Association's award for medical reporting. His work has appeared in National Geographic, Newsweek, Hippocrates, and many other national magazines. He is also the author of In Self-Defense (Harcourt Brace Jovanovich), Active Living Every Day, and Heart Healthy for Life.

References

First published October 28, 2003
Last updated October 30, 2007
Copyright © 2003 Consumer Health Interactive

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