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ACTIVE NOT RECRUITING
NCT04613128

The PROMISE Pediatric Study 6 to 11 Years Old

Sponsor: Nicole Hamblett

View on ClinicalTrials.gov

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