Clinical trials

What is a clinical trial?

A clinical trial is a study where the effectiveness of a treatment is tested in a group of people, e.g. people with pulmonary hypertension (PH).

Why are clinical trials important?

Clinical trials play a very important role in the development of new treatments for different diseases and conditions. Without clinical trials there would have been no developments in new treatments for PH.

Why participate in a clinical trial?

Clinical trials could help you get a new treatment for PH before it is approved. You will also be helping improve medical advances in the treatment of PH.

PH is a relatively rare disease. This means that it may be difficult for drugs companies to get enough people involved in a clinical trial and can delay new treatments becoming available.

Here are some things to think about before you enrol on a clinical trial for PH:

• Your PH may improve during a clinical trial, but it may stay the same.
• The number of clinic visits may be more than the amount of times you normally attend.
• All the drugs that are tested in people with a condition such as PH have already been tested in healthy volunteers. The risk of serious side effects are extremely low.
• You may be given a placebo (dummy drug) during the trial and not the drug being tested.
• Some of your blood samples may be kept after the clinical trial ends (with your consent).

If you want to enrol on a clinical trial, your PH team can provide you with details of clinical trials relevant to you.

Clinical trials and PH

There are several new drug treatments in early clinical trials, or about to start clinical trials, specifically in people with PH. Trials are ongoing to investigate selexipag and it’s relative ralenipag. There is also great progress being made in genetic engineering, hoping to find a whole new generation of PH treatments. One treatment being tested is a drug called imatinib, which is known as an anti-growth factor. It is already used to treat some types of cancer. Clinical trials of imatinib for people with PH are ongoing.