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The PROMISE Pediatric Study 6 to 11 Years Old
Sponsor: Nicole Hamblett
Summary
This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor (ETI) triple combination therapy in children (6-11 years of old) with one or more copies of the F508del mutation, study the effects of ETI across a number of CF disease manifestations, and collect specimens for future research. Subjects in the study will have one "before ETI" visit within 30 days before initiation of the therapy and five "after ETI" visits over a 24-month follow-up period. Participants who have participated in the original PROMISE Pediatric Sub-Study have the option of participating in a long-term extension with annual visits performed at the 36- and 48-month timepoints. The durability of the clinical and biological changes in the PROMISE Pediatric Sub-Study can be assessed with extended follow-up, which would enable the sub-studies to consider potential clinical consequences of the biological or physiological effects being studied. This work will help to inform long term prognosis and feasibility of certain clinical trials outcomes for interventional studies and may be useful when considering research priorities in drug development. The duration of participation for each subject is 24 months (with an additional 24 months if participants agree to the optional long-term extension). NOTE: FDA has granted approval for elexacaftor, tezacaftor and ivacaftor in the 6-11 age group.
Official title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (the PROMISE Pediatric 6 to 11 Years Old Study)
Key Details
Gender
All
Age Range
6 Years - 11 Years
Study Type
OBSERVATIONAL
Enrollment
180
Start Date
2021-06-11
Completion Date
2026-01-31
Last Updated
2025-11-03
Healthy Volunteers
No
Locations (20)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Stanford University Medical Center
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
John Hopkins University
Baltimore, Maryland, United States
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, United States
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, United States
Children's Mercy Kansas City
Kansas City, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
University of North Carolina at Chapel Hill
Chapel Hill, 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
Oregon Health Sciences University
Portland, Oregon, United States
Baylor College of Medicine
Houston, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
University of Wisconsin
Madison, Wisconsin, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States