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Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
NCT04509050 · View on ClinicalTrials.gov ↗
Study Summary
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
Conditions Studied
Interventions
- DRUG Ivacaftor or elexacaftor/tezacaftor/ivacaftor
Study Locations (20)
New York
- The Cystic Fibrosis Center of Western New York — Buffalo
- Children's Hospital of New York — New York
- SUNY Upstate Medical University — Syracuse
- New York Medical College at Westchester Medical Center — Valhalla
Florida
- Nemours Children's Clinic — Jacksonville
- University of Miami — Miami
- The Nemours Children's Clinic - Orlando — Orlando
Michigan
- University of Michigan, Michigan Medicine — Ann Arbor
- Helen DeVos Children's Hospital — Grand Rapids
Minnesota
- Children's Hospitals and Clinics of Minnesota — Minneapolis
- The Minnesota Cystic Fibrosis Center — Minneapolis
Missouri
- Children's Mercy Kansas City — Kansas City
- St. Louis Children's Hospital — St Louis
Alabama
- The Children's Hospital Alabama, University of Alabama at Birmingham — Birmingham
California
- Stanford University Medical Center — Palo Alto
Colorado
- Children's Hospital Colorado — Aurora
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 210 participants |
| Start Date | 2020-11-18 |
| Est. Completion | 2029-12-01 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04509050
The ClinicalTrials.gov registry entry for NCT04509050 describes a study currently listed as 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 210 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sonya Heltshe, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Cystic Fibrosis appearing as the primary indexed condition, and to 1 intervention — of which Ivacaftor or elexacaftor/tezacaftor/ivacaftor is the first listed. 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: NCT04509050 reports 20 study locations spanning 12 distinct geographic areas — top geographies include New York, Florida, Michigan. 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 NCT04509050 about?
NCT04509050 is a clinical study titled "Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children". This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
What is the current status of trial NCT04509050?
This trial is currently recruiting. The enrollment target is 210 participants. The study started on 2020-11-18. Estimated completion is 2029-12-01.
What conditions does trial NCT04509050 study?
This clinical trial studies the following conditions: Cystic Fibrosis. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04509050?
The interventions under investigation include: Ivacaftor or elexacaftor/tezacaftor/ivacaftor (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04509050?
This trial is sponsored by Sonya Heltshe, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04509050 being conducted?
This trial has 20 study locations across Alabama, California, Colorado, Florida, Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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