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The PROMISE Pediatric Study 6 to 11 Years Old
NCT04613128 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Study Locations (20)
Missouri
- Children's Mercy Kansas City — Kansas City
- Washington University School of Medicine — St Louis
Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati
- Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center — Cleveland
Wisconsin
- University of Wisconsin — Madison
- Children's Hospital of Wisconsin — Milwaukee
Alabama
- University of Alabama at Birmingham — Birmingham
California
- Stanford University Medical Center — Palo Alto
Colorado
- Children's Hospital Colorado — Aurora
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
Indiana
- Riley Hospital for Children — Indianapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 180 participants |
| Start Date | 2021-06-11 |
| Est. Completion | 2026-01-31 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04613128
The ClinicalTrials.gov registry entry for NCT04613128 describes a study currently listed as active not recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 180 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Nicole Hamblett, which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Cystic Fibrosis appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04613128 reports 20 study locations spanning 17 distinct geographic areas — top geographies include Missouri, Ohio, Wisconsin. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04613128 about?
NCT04613128 is a clinical study titled "The PROMISE Pediatric Study 6 to 11 Years Old". 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 acr...
What is the current status of trial NCT04613128?
This trial is currently active not recruiting. The enrollment target is 180 participants. The study started on 2021-06-11. Estimated completion is 2026-01-31.
What conditions does trial NCT04613128 study?
This clinical trial studies the following conditions: Cystic Fibrosis, Cystic Fibrosis in Children. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT04613128?
This trial is sponsored by Nicole Hamblett, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04613128 being conducted?
This trial has 20 study locations across Alabama, California, Colorado, Illinois, Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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