NCT04509050 Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
| NCT ID | NCT04509050 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Sonya Heltshe |
| Condition | Cystic Fibrosis |
| Study Type | OBSERVATIONAL |
| Enrollment | 210 participants |
| Start Date | 2020-11-18 |
| Primary Completion | 2029-12-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 210 participants in total. It began in 2020-11-18 with a primary completion date of 2029-12-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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).
Eligibility Criteria
Inclusion Criteria: * Part A: * Less than 10 years of age at the first study visit. * Documentation of a CF diagnosis. Part B: * Participated in Part A OR less than 7 years of age at the first study visit. * Documentation of a CF diagnosis. * CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor). * Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor. Exclusion Criteria: * Part A and Part B: * Use of an investigational drug within 28 days prior to and including the first study visit. * Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit. * Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.
Contact & Investigator
Bonnie Ramsey, MD
PRINCIPAL INVESTIGATOR
Seattle Children's Hospital
Frequently Asked Questions
Who can join the NCT04509050 clinical trial?
This trial is open to participants of all sexes, up to 10 Years, studying Cystic Fibrosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04509050 currently recruiting?
Yes, NCT04509050 is actively recruiting participants. Contact the research team at rachael.buckingham@seattlechildrens.org for enrollment information.
Where is the NCT04509050 trial being conducted?
This trial is being conducted at Birmingham, United States, Palo Alto, United States, Aurora, United States, Jacksonville, United States and 11 additional locations.
Who is sponsoring the NCT04509050 clinical trial?
NCT04509050 is sponsored by Sonya Heltshe. The principal investigator is Bonnie Ramsey, MD at Seattle Children's Hospital. The trial plans to enroll 210 participants.