The PROMISE Pediatric Study 6 to 11 Years Old
Public ClinicalTrials.gov record NCT04613128. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04613128
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 125 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- 6 Years to 11 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 10, 2021
- Primary completion
- Dec 7, 2025
- Completion
- Dec 7, 2025
- Last update posted
- Apr 26, 2026
2021 – 2025
United States locations
- U.S. sites
- 20
- U.S. states
- 17
- U.S. cities
- 20
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294 | — |
| Stanford University Medical Center | Palo Alto | California | 94304 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| John Hopkins University | Baltimore | Maryland | 21287 | — |
| Boston Children's Hospital, Brigham & Women's Hospital | Boston | Massachusetts | 02115 | — |
| The Minnesota Cystic Fibrosis Center | Minneapolis | Minnesota | 55455 | — |
| Children's Mercy Kansas City | Kansas City | Missouri | 64108 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44146 | — |
| Oregon Health Sciences University | Portland | Oregon | 97239 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| Virginia Commonwealth University | Richmond | Virginia | 23219 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
| University of Wisconsin | Madison | Wisconsin | 53792 | — |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04613128, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 26, 2026 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04613128 live on ClinicalTrials.gov.