NCT04613128Completed
The PROMISE Pediatric Study 6 to 11 Years Old
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (the PROMISE Pediatric 6 to 11 Years Old Study)
Source: ClinicalTrials.gov · Last updated 27 Apr 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 125 (actual)
- First submitted
- 27 Oct 2020
- Start date
- 11 Jun 2021
- Primary completion date
- 8 Dec 2025
- Lead sponsor
- Nicole Hamblett
- Diseases
- Cystic fibrosis
- NCT number
- NCT04613128
What you commit to
- Study type: Observational
- Participants enrolled: 125
- From 11 Jun 2021 to the primary completion date, 8 Dec 2025
- 20 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- 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.
- History of lung or liver transplantation,or listing for organ transplantation.
Who the study is looking for
- 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).
Age in the registry: 6 years to 11 years · Sex: All · Does not accept healthy volunteers
Sites (20)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Hospital Colorado Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| John Hopkins University Coordinator of this site? Confirm its status | Baltimore | United States | Unknown status |
| University of Alabama at Birmingham Coordinator of this site? Confirm its status | Birmingham | United States | Unknown status |
| Boston Children's Hospital, Brigham & Women's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| University of North Carolina at Chapel Hill Coordinator of this site? Confirm its status | Chapel Hill | United States | Unknown status |
| Ann & Robert H. Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| Cincinnati Children's Hospital Medical Center Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Rainbow Babies and Children's Hospital / University Hospitals Cleveland Medical Center Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| Baylor College of Medicine Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| Riley Hospital for Children Coordinator of this site? Confirm its status | Indianapolis | United States | Unknown status |
| University of Iowa Coordinator of this site? Confirm its status | Iowa City | United States | Unknown status |
| Children's Mercy Kansas City Coordinator of this site? Confirm its status | Kansas City | United States | Unknown status |
| University of Wisconsin Coordinator of this site? Confirm its status | Madison | United States | Unknown status |
| Children's Hospital of Wisconsin Coordinator of this site? Confirm its status | Milwaukee | United States | Unknown status |
| The Minnesota Cystic Fibrosis Center Coordinator of this site? Confirm its status | Minneapolis | United States | Unknown status |
| Stanford University Medical Center Coordinator of this site? Confirm its status | Palo Alto | United States | Unknown status |
| Oregon Health Sciences University Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| Virginia Commonwealth University Coordinator of this site? Confirm its status | Richmond | United States | Unknown status |
| Seattle Children's Hospital Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
| Washington University School of Medicine Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.