A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT06504589
- Lead Sponsor
- Nicole Hamblett
- Brief Summary
The REACH study is for people with CF who do not take cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The goal of the REACH study is to collect research data, including health data and specimens, from people with CF who do not take CFTR modulators. This data may be used to inform CF research, help design CF clinical trials and support the development of new treatments for people with CF who do not take CFTR modulators.
Another goal of this study is to learn about research involvement for people with CF who do not take CFTR modulators, engage them in research, and give them an opportunity to learn about what is involved in participating in a CF research study.
- Detailed Description
People with Cystic Fibrosis (pwCF) who are genetically ineligible and/or not taking cystic fibrosis transmembrane conductance regulator (CFTR) modulators currently face future health that is considerably different from the approximately 90% of pwCF in the United States who benefit from CFTR modulators. New treatments are being advanced for pwCF who are genetically ineligible or not taking CFTR modulators and these therapies will include both nucleic acid-based therapies (NABTs) to address the underlying defect in CFTR and symptomatic therapies aimed at targeting the symptoms of CF. A key concern for this limited and underserved patient population is being able to advance multiple therapeutic programs in parallel. To complete these studies, CF researchers will need to be able to reach this community effectively while also promoting the use of innovative trial designs.
The REACH Study is a prospective, longitudinal, observational research study to obtain research quality (i.e., monitored research) CF outcome data. Primary outcome endpoints of the Core study (collected across all study participants) are aligned with anticipated clinical trial outcome endpoints needed in overall development of therapies for the CF population unable to benefit from CFTR modulators. This study will also include sub-studies to obtain specialized measures which may help inform efficacy and safety evaluations of new therapies by providing CF control data. Finally, this study also seeks to assess research solicitation and research participation for the CF population that is modulator ineligible or not taking modulators. The observational data collected within this study may be used in characterizing this CF population, developing innovative trial designs, for comparison when evaluating new or experimental CF therapies, and/or in CF research.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 400
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in ppFEV1 at 12 months 12 months Mean change (standard deviation) in percent predicted forced expiratory volume in 1 second (ppFEV1) as measured by in-clinic spirometry from baseline to 12 months post-baseline.
- Secondary Outcome Measures
Name Time Method Change in LCI 5% at 12 months 12 months Mean change (standard deviation) in lung clearance index (LCI) calculated as 1/20th of starting end-tidal concentration of N2 (LCI 5%) as measured by multiple breath washout (MBW) from baseline to 12 months post-baseline.
Change in CFQ-R-RD at 12 months 12 months Mean change (standard deviation) in the Cystic Fibrosis Question - Revised Respiratory Domain (CFQ-R-RD) from Baseline to 12 months post-baseline. Scores for the CFQ-R-RD range from 0 to 100.
Change in LCI 2.5% at 12 months 12 months Mean change (standard deviation) in lung clearance index (LCI) calculated as 1/40th of starting end-tidal concentration of N2 (LCI 2.5%) as measured by multiple breath washout (MBW) from baseline to 12 months post-baseline.
Absolute 12-month change in MCC Index 12 months Mean change (standard deviation) in mucociliary clearance (MCC) index as by the MCC procedure per gamma scintigraphy from baseline to 12 months post-baseline.
Change in home spirometry ppFEV1 at 12 months 12 months Mean change (standard deviation) in percent predicted forced expiratory volume in 1 second (ppFEV1) as measured by home spirometry from baseline to 12 months post-baseline.
Relative 12-month change in MCC Index 12 months Mean change (standard deviation) in mucociliary clearance (MCC) index as by the MCC procedure per gamma scintigraphy from baseline to 12 months post-baseline divided by the baseline MCC index.
Trial Locations
- Locations (68)
The Children's Hospital Alabama, University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Tucson Cystic Fibrosis Center
🇺🇸Tucson, Arizona, United States
University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States
University of California San Diego
🇺🇸La Jolla, California, United States
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Stanford University Medical Center
🇺🇸Palo Alto, California, United States
University of California, San Francisco - Adult Center
🇺🇸San Francisco, California, United States
University of California, San Francisco - Peds Center
🇺🇸San Francisco, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Florida
🇺🇸Gainesville, Florida, United States
University of Miami
🇺🇸Miami, Florida, United States
Central Florida Pulmonary Group
🇺🇸Orlando, Florida, United States
Tampa General Hospital
🇺🇸Tampa, Florida, United States
Children's Healthcare of Atlanta and Emory University
🇺🇸Atlanta, Georgia, United States
Augusta University
🇺🇸Augusta, Georgia, United States
Saint Luke's Cystic Fibrosis Center of Idaho
🇺🇸Boise, Idaho, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Tulane University
🇺🇸Metairie, Louisiana, United States
John Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Massachusetts Memorial Health Care
🇺🇸Worcester, Massachusetts, United States
University of Michigan, Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
Wayne State University Harper University Hospital
🇺🇸Detroit, Michigan, United States
Corewell Health Helen DeVos
🇺🇸Grand Rapids, Michigan, United States
The Minnesota Cystic Fibrosis Center
🇺🇸Minneapolis, Minnesota, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Billings Clinic
🇺🇸Billings, Montana, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
The Cystic Fibrosis Center of Western New York
🇺🇸Buffalo, New York, United States
Cohen Children's Medical Center of New York
🇺🇸Lake Success, New York, United States
Columbia University Cystic Fibrosis Program
🇺🇸New York, New York, United States
University of Rochester Medical Center Strong Memorial
🇺🇸Rochester, New York, United States
New York Medical College at Westchester Medical Center
🇺🇸Valhalla, New York, United States
University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Atrium Health Wake Forest Baptist
🇺🇸Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
Cleveland Clinic Cystic Fibrosis Program
🇺🇸Cleveland, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Dayton Children's Hospital
🇺🇸Dayton, Ohio, United States
Oregon Health & Sciences University
🇺🇸Portland, Oregon, United States
Hershey Medical Center Pennsylvania State University
🇺🇸Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
🇺🇸Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Prisma Health Children's Hospital - Midlands
🇺🇸Columbia, South Carolina, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Dell Children's Medical Center of Central Texas
🇺🇸Austin, Texas, United States
University of Texas Southwestern / Children's Health
🇺🇸Dallas, Texas, United States
University of Texas Southwestern
🇺🇸Dallas, Texas, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
Adult Cystic Fibrosis Center at the University of Utah
🇺🇸Salt Lake City, Utah, United States
Inova L.J. Murphy Pediatric CF Program
🇺🇸Fairfax, Virginia, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
Providence Medical Group, Cystic Fibrosis Clinic
🇺🇸Spokane, Washington, United States
West Virginia University - Morgantown
🇺🇸Morgantown, West Virginia, United States
University of Wisconsin
🇺🇸Madison, Wisconsin, United States