The PROMISE Pediatric Study 6 to 11 Years Old

Completed

Conditions studied: Cystic Fibrosis, Cystic Fibrosis in Children

In brief

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.

Key facts

Study ID: NCT04613128
Run by: Nicole Hamblett
People needed: 125
Starts: 2021-06-11
Expected to finish: 2025-12-08
Last updated by the study team: 2026-04-27

Who can join

Age: 6 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Written parental informed consent and assent obtained from subject and the subject's legal guardian.
Be willing and able to adhere to the study visit schedule and other protocol requirements.
All genders 6-11 years old on Day 1.
Diagnosis of CF.
CFTR mutations consistent with the FDA labeled indication for the ETI.
Physician intent to prescribe the ETI.
Able to attempt the testing and procedures required for this study, as judged by the investigator.
Enrolled in the Cystic Fibrosis Foundation Patient Registry.
Clinically stable with no significant changes in health status within the 14 days prior to Visit 1 (and inclusive of Visit 1).

You may not qualify if…

Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
Use of any ETI within the 180 days prior to Visit 1.
Any acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids within the 14 days prior to Visit 1 (inclusive of Visit 1) for lower respiratory tract symptoms.
Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, inhaled tobramycin, Cayston®, Kalydeco, Orkambi®, Symdeko®) within the 4 weeks prior to Visit 1 (inclusive of Visit 1).
Use of an investigational agent within the 28 days prior to Visit 1.
Use of chronic oral corticosteroids (equivalent to 10 mg. or more per day of prednisone) within the 28 days prior to Visit 1.
Treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled) within the 28 days prior to Visit 1.
History of lung or liver transplantation,or listing for organ transplantation.

Where it is running

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

Full record on ClinicalTrials.gov

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