What is a clinical trial?

A clinical trial is a type of research study involving patients. A clinical trial evaluates whether a new type of lab test, x-ray, or treatment may be helpful in patients.

Most clinical trials in patients with PH will test the effects of a new medication for PH. Effects of PH medications include the helpful, beneficial effects on patients, as well as possible side-effects and risks.

Clinical research trials of PH treatments are usually carried out by companies that make PH treatments, known as sponsors. These sponsors will provide the PH treatment free of charge to you, in exchange for information about your PH, your symptoms, and the effects the treatment has on you.

Most clinical research trials of PH treatments will randomly assign patients to different treatment groups. You may be given the actual PH treatment, or you may receive a sugar pill (also known as placebo). You will not know which treatment you receive. Your doctor and nurse also may not know which treatment you are receiving. This refers to a double-blind procedure.

If a new lab test or treatment is found to be safe and effective in PH patients in a clinical trial, then it can eventually be offered to other PH patients.

It is important to realize that the tests and treatments that are used for PH today were developed and tested in clinical trials, before they were available for PH patients. This type of research is critical to the development of new treatments for PH.