Evaluating the effects of CFTR modulators in young children with cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to explore the biological and clinical effects of CFTR modulators in infants and young children diagnosed with cystic fibrosis. It consists of two parts: the first part will track the natural history of hormonal growth factors and assess feasibility over a period of up to five years, while the second part will observe the impact of specific CFTR modulators on growth over a 60-month follow-up. Participants will undergo multiple visits to monitor various health parameters, including lung function and gastrointestinal health. The study is designed to provide insights into the long-term effects of these therapies in a pediatric population.

Why it matters

Who can join: the full rules

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.

Where it's running

• The Children's Hospital Alabama, University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• Stanford University Medical Center
  Palo Alto, California, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• Nemours Children's Clinic
  Jacksonville, Florida, United States Recruiting
• University of Miami
  Miami, Florida, United States Recruiting
• The Nemours Children's Clinic - Orlando
  Orlando, Florida, United States Recruiting

Study contacts

• Principal investigator: Bonnie Ramsey, MD — Seattle Children's Hospital
• Study coordinator: Rachael Buckingham
• Email: rachael.buckingham@seattlechildrens.org
• Phone: 206-884-7517

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.