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NCT04509050 · ClinicalTrials.gov registry record
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
A clinical trial of Cystic Fibrosis, sponsored by Sonya Heltshe.
- Recruiting
- Registry status
- 210
- Enrollment target
- 20
- Study locations
NCT04509050: Recruiting study of Cystic Fibrosis, sponsored by Sonya Heltshe.
NCT04509050 is a study of Cystic Fibrosis that is actively recruiting participants, run by Sonya Heltshe. The registered enrollment target is 210 participants, above the 178-participant average among 169 other Cystic Fibrosis trials with a reported enrollment target (18% higher). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04509050, a study of Cystic Fibrosis, is actively recruiting participants, sponsored by Sonya Heltshe.
- RECRUITING
- Registry status
- 210 participants
- Enrollment target
- 20
- Study locations
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).
Primary Outcome
Weight-for-age z-scores over time
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 |
What NCT04509050 shows while recruiting
NCT04509050 is an observational study that tracks outcomes without assigning an intervention. The registered 210 participants enrollment target is mid-sized for trials with a published cap, above the 178-participant average among 169 other Cystic Fibrosis trials with a reported enrollment target (18% higher).
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.
NCT04509050 names 20 study sites across 12 states, led by New York, Florida, Michigan.
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.
What interventions are being tested in trial NCT04509050?
The interventions under investigation include: Ivacaftor or elexacaftor/tezacaftor/ivacaftor (DRUG).
Who is sponsoring clinical trial NCT04509050?
This trial is sponsored by Sonya Heltshe, which has 1 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Cystic Fibrosis
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04509050's enrollment target sits among peer trials
210 34th of 169 higher than 135 of 169 other Cystic Fibrosis trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Cystic Fibrosis trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
RECRUITING
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Assessing Tenapanor as a Treatment of CF-related Constipation.
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Genetics of Insulin and Incretins in Cystic Fibrosis
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