Yes! Please visit the ‘Clean PCD Trial‘ page for more information.
Research in PCD falls into two broad categories: 1) Basic science, and 2) Clinical research. Both are critically important in finding a cure for PCD. Basic science does not involve human beings, although it may rely on human tissue or cells. Clinical research refers to any type of study or trial that involves actual people. The PCD community has been so small and so geographically dispersed that it has been challenging to conduct clinical research—until now. By collaborating with PCD investigators and patient groups around the world, the international PCD community will now have the opportunity to participate in research aimed at better understanding the disorder (e.g. long term—or ‘longitudinal’ observational studies of the natural history of PCD), determining whether existing therapies used in other disorders will work for PCD (e.g. azithromycin trial in Europe), identifying potential new therapies (e.g. CLEAN-PCD trial in North America and Europe). With a growing patient community and growing interest on the part of researchers and drug developers, we anticipate that there will be many other research opportunities in the near future.
Having a verified diagnosis, especially knowing your genetics, will be very important in PCD research trials going forward. It is also important to note that number of years aside, PCD is associated with significant illness and quality of life deficits throughout the lifespan. This is why research that will lead to better diagnosis, treatments and cures are so critical for the population.