CLEAN-PCD Trial

ANNOUNCING A CLINICAL TRIAL OPPORTUNITY FOR INDIVIDUALS WITH PCD:

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 the official Clinical Trials site.

 

Rationale

In PCD, ineffective ciliary function leads to stagnant mucus in the airways. Retained mucus is a breeding ground for infectious pathogens. Infection and inflammation in the airways triggers additional mucus production and the mucus becomes thicker and harder to remove. This creates a ‘vicious cycle’ of mucus hypersecrection, infection, inflammation and more mucus hypersecretion. The proposed mechanism of this investigational compound VX-371 is reducing the burden of this vicious cycle through more effective clearance of mucus.

 

Criteria

To preserve the integrity of the data collected during the CLEAN-PCD trial, there are carefully selected inclusion (who can participate) and exclusion (who cannot) criteria for this study. Clinical studies seek to answer a clinical question(s) and to ensure that these questions are the only focus, careful attention is paid to prevent ‘confounding’ issues from arising. Inclusion and exclusion criteria for CLEAN-PCD can be found at the link below.

Please note that there are 35 centers in 8 countries participating in this trial so far and individuals who wish to participate will need to be seen at one of these centers.

 

Study Design

The study design for CLEAN-PCD ensures that every participant will be on the investigational drug at some point during the trial. There will be two phases–one on drug or placebo, followed by a washout period, and then the second phase where participants will receive either the drug or placebo they did not get in phase I.

 

Goal

The goal of research is to provide generalized knowledge that can be applied to a population under study. This is different than a therapeutic relationship with your own doctor, which is designed to help you as an individual. You may not personally benefit from participating in research. Choosing not to participate in a clinical trial will not affect your relationship with your healthcare provider or with the PCDF. It is a strictly personal decision.

 

*Study Update*

The following sites are now activated for screening patients. If you are interested in participating in the study at any of these sites, please contact the Vertex patient line or the Parion study coordinator:

Medical Information 617-341-6777 / medicalinfo@vrtx.com

Ashley Simmons: 919-313-1210 / asimmons@parion.com

Check back frequently for updates. A map with all participating sites is posted below.

DISCLAIMER: Information about the CLEAN-PCD study is provided for information only and does not constitute an endorsement by the PCDF.

Alabama

George Solomon, MD

University of Alabama Birmingham

Birmingham, AL


California

Carlos Milla, MD & Jackie Zirbes, RN

Stanford University

Palo Alto, CA


Colorado

Scott Sagel, MD & Carol Kopecky, RN

Colorado Children’s

Aurora, CO


Florida

Mattias Salathe, MD & Patricia Graham, RN

University of Miami

Miami, FL

Marisa Couluris, MD

University of South Florida/Morsani College of Medicine

Tampa, FL


Kansas

Hugo Escobar, MD & Candy Schmoll, RN, BSN, CCRC

Children’s Mercy Hospital

Kansas City, MO

Deepika Polineni, MD

University of Kansas

Kansas City, KS


Illinois

Mary Nevin, MD

Lurie Children’s Hospital

Chicago, IL

Michelle Prickett, MD

Northwestern Memorial Hospital

Chicago, IL


Indiana

Stephanie Davis, MD

Riley Hospital fro Children at Indiana University Health

Indianapolis, IN


Iowa

Douglas Hornick, MD

University of Iowa

Iowa City, IA


Massachusetts

Kenan Haver, MD

Boston Children’s Hospital

Boston, MA


Michigan

Thomas Saba, MD

University of Michigan

Ann Arbor, MI


Minneapolis

Anne Griffiths, MD

Children’s Minnesota Research

Minneapolis, MN


Missouri

Thomas Ferkol, Jr, MD

Washington University in St. Louis

St. Louis, MO


New York

Angela Dimango, MD

Columbia University Medical Center – New York Presbyterian Hospital

New York, NY


North Carolina

Margaret Leigh, MD

University of North Carolina at Chapel Hill

Chapel Hill, NC


Ohio

Steven Strausbaugh, MD & Laura Veri, RN

UHCMC/Rainbow Babies & Children’s Hospital

Cleveland, OH


Ontario

Sharon Dell, MD

Hospital for Sick Children

Toronto, Ontario


South Carolina

Trey Brown, MD

USC Pediatric Research

Columbia, SC


Washington DC

Anne O’Donnell, MD

MedStar Georgetown University Hospital

Washington, DC


Washington State

Margaret Rosenfeld, MD

Seattle Children’s

Seattle, WA


Quebec

Adam Shapiro, MD

Health Centre for Innovative Medicine

Montreal, Quebec

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