Creating a Research Infrastructure
The PCDF was established to support and accelerate research for individuals with PCD, reflecting our belief that the best way to support the patient community is to ensure they have access to appropriate therapies and accurate information to inform care decisions, aid in family planning and improve quality of life. The information required to meet these ambitious goals will come from adequate and well‐controlled clinical trials. For this reason, we strive to be a ‘research‐focused’ organization.
As a small patient advocacy organization, our direct investment is research is limited. To leverage limited resources we focus on strategic partnerships with researchers, professional societies and private funders. Additionally, we provide support for scientific meetings and travel awards for young investigators.
The Path to Clinical Trials
The Path to Clinical Trials (PTCT) is the main focus of our Research Support Program. The PTCT consists of two critical infrastructure components needed to support clinical trial participation in PCD:
PCDF Clinical and Research Centers Network (PCD‐CRCN or just CRCN)
The PCDF CRNC is modeled after the highly successful Cystic Fibrosis Foundation Care Centers. Each multi‐disciplinary site is required to meet stringent criteria for the diagnosis of PCD and to follow PCDF Consensus guidelines published in Pediatric Pulmonology in 2015 (attached). The sites undergo an accreditation visit and are evaluated by PCDF personnel and by clinical site reviewers. The CRNC currently has 29 sites in various stages of accreditation.
North American PCD Registry (NAPCDR)
Our primary focus in the immediate terms is funding the NAPCDR. The NAPCDR is modeled after the Cystic Fibrosis Registry, with the goal of collecting reliable core data from our clinical sites. Additionally, we hope to incorporate a patient‐report component to supplement and inform the core clinical data. The need for reliable clinical data on PCD is critical and finding resources to support the development of the NAPCDR is a top priority.
Moving the Science Forward
‘PCD on the Move’ Annual Scientific Conference
Our annual scientific conference is another component of our Research program. This meeting brings together top PCD experts from around the world to discuss cutting edge PCD basic and clinical sciende research. It also serves as a training meeting for new investigators and clinicians.
Funding Quality Research
Strategic Research Partnerships
ATS/PCDF Partner GrantsThe PCDF partners annually with the American Thoracic Society’s (ATS) Partner Grant program. This matching grant partnership allows us to maximize our research dollars while ensuring that our funds are directed to high-quality projects. For more information about this program, click here. To see a list of our current grantees, please click here.
Genetic Disorders of Mucociliary Clearance Consortium
The Genetic Disorders of Mucociliary Clearance Consortium, or GDMCC, is a member consortium of the Rare Diseases Clinical Research Network (RDCRN), an initiative of the Office of Rare Diseases Research (ORDR) and the National Center for Advancing Translational Science (NCATS) at NIH.
The GDMCC is a network of nine North American Centers that are collaborating in diagnostic testing, genetic studies, and clinical trials in patients with impairments in mucociliary clearance, focusing on primary ciliary dyskinesia, cystic fibrosis, and pseudohypoaldosteronism. Additionally, GDMCC studies target related clinical conditions believed to be due to impaired mucociliary clearance, including idiopathic bronchiectasis and infection with non-tuberculous mycobacterial (NTM) organisms. Ultimately, we hope to better define the clinical pathogenesis of these important airway diseases, improve or expand diagnostic testing, and develop new and effective treatments.
The GDMCC is supported by ORDR/NCATS and by the National Heart, Lung and Blood Institute (NHLBI). Click here for more information.
Current Clinical Trials
CLEAN-PCD Clinical Trial
Parion Sciences and Vertex Pharmaceuticals announce the CLEAN-PCD trial of a novel inhaled compound (VX-371) designed to improve airway hydration and aid with clearance of mucus. CLEAN-PCD, an international clinical study, will evaluate the effectiveness of this compound in individuals with PCD. For more information about this particular study, please visit to the official Clinical Trials site.
For more information on currently open GDMCC protocols, please check the GMDCC site here.
Additional PCD study opportunities can be found at ClinicalTrials.gov.