A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)
- Conditions
- Cystic Fibrosis
- Interventions
- Other: Observational
- Registration Number
- NCT02477319
- Lead Sponsor
- University of Alabama at Birmingham
- Brief Summary
identify and validate biomarkers that might reflect partial restoration of CFTR function and can be used to monitor disease progression, and ii) evaluate the mechanistic effects of CFTR modulators and other relevant therapies in individuals with CF
- Detailed Description
Cystic fibrosis (CF) is a genetic disorder caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Over 1,900 mutations, categorized into five genotypic or functional classes are implicated in causing CF. Severity of disease varies widely in CF based on CFTR-dependent and independent factors. Progressive obstructive lung disease is the main determinant of morbidity and mortality in CF; therefore it is critical to identify biomarker profiles that reflect and predict this phenotypic variability, and understand their relationship to residual CFTR activity. Emerging CFTR modulator therapies that directly target defective CFTR are being evaluated in pivotal clinical trials and may become available in the next few years. It is not known how partial restoration of CFTR function might impact CF disease progression and disease-related biomarkers. Thus there is urgent need to i) identify and validate biomarkers that might reflect partial restoration of CFTR function and can be used to monitor disease progression, and ii) evaluate the mechanistic effects of CFTR modulators and other relevant therapies in individuals with CF
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 452
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Part A Observational * Cohort 1: Healthy Controls * Cohort 2: Partial CFTR function CF (class IV/V) * Cohort 3: Absent CFTR function CF (Class I/II) Part B Observational CF patients who are homozygous for the F508del
- Primary Outcome Measures
Name Time Method Sweat Chloride by Cohort (Part A Only) For cohort 1, sweat chloride at Day 0 is time frame. For cohorts 2-3, sweat chloride averaged across all 3 visits at days 0, 14 and 90 is time frame. This is the primary endpoint for Part A per the PROSPECT protocol. Mean sweat chloride was not reported for Part B, as it is not a relevant statistic.
For cohort 1, sweat chloride is from day 0 only. For cohorts 2-3, sweat chloride was averaged from days 0, 14, 90 via a random intercept longitudinal model.6 Month Change in FEV1 Percent Predicted (Part B Only) Baseline and 6 months This is the primary endpoint for Part B per the PROSPECT protocol. Change in FEV1 Percent Predicted is only relevant for Part B as it captures changes in lung function post-initiation of Ivacaftor/Lumacaftor.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (38)
Children's Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Hershey Medical Center; Penn State Children's Hospital
🇺🇸Hershey, Pennsylvania, United States
Froedtert Hospital
🇺🇸Milwaukee, Wisconsin, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Indianapolis University Hospital; James Whitcomb Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
John Hopkins University
🇺🇸Baltimore, Maryland, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
University Hospital of Cleveland
🇺🇸Cleveland, Ohio, United States
Baylor College of Medicine/Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Nation Wide Childrens Hospital
🇺🇸Columbus, Ohio, United States
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Lucile S. Packard Children's Hospital
🇺🇸Palo Alto, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
The University of Kansas Hospital
🇺🇸Kansas City, Kansas, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Children's Hospital Boston
🇺🇸Boston, Massachusetts, United States
Devon Children's Hospital at Spectrum Health
🇺🇸Grand Rapids, Michigan, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Cardinal Glennon Children's Medical Center
🇺🇸Saint Louis, Missouri, United States
Dartmouth Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Women and Children's Hospital of Buffalo
🇺🇸Buffalo, New York, United States
Maria Fareri Children's Hospital; Westchester Medical Center
🇺🇸Valhalla, New York, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
Children's Hospital of Michigan
🇺🇸Detroit, Michigan, United States
Oregon Health & Sciences University
🇺🇸Portland, Oregon, United States
The Children's Hospital at Vanderbilt
🇺🇸Nashville, Tennessee, United States
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
The Children's Hospital Colarado
🇺🇸Aurora, Colorado, United States