Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

Recruiting

• Conditions: Cystic Fibrosis
• Interventions: Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor

• Registration Number: NCT04509050
• Lead Sponsor: Sonya Heltshe

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).

Detailed Description

This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact in children with cystic fibrosis (CF) on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health.

Total duration of the study is expected to be 10 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth factors in early childhood and assess the feasibility of additional measurements. In Part A, subjects will have up to 8 visits over a period of up to 5 years.

Part B will be a prospective longitudinal study to observe the effects of administration of either ivacaftor or elexacaftor/tezacaftor/ivacaftor (elex/tez/iva) on growth. In Part B, subjects will have 1 "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and 8 "after ivacaftor or elex/tez/iva" visits over a 60-month follow-up period.

Study Timeline

• Study First Submitted: 2020-08-06
• Study First Submitted QC: 2020-08-10
• Study First Posted: 2020-08-11
• Study Start: 2020-11-18
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-08
• Last Update Posted: 2024-08-12
• Primary Completion: 2029-12-01
• Study Completion: 2029-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 210

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Part B	Ivacaftor or elexacaftor/tezacaftor/ivacaftor	Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the Part A cohort of this study may enroll into the Part B cohort if they become eligible for these CFTR modulator therapies and plan to start them.

Primary Outcome Measures

Name	Time	Method
Part A Primary Outcome Measure: Change in weight-for-age z-scores	Baseline to 12 months	Weight-for-age z-scores over time
Part A Primary Outcome Measure: Change in height-for-age z-scores	Baseline to 12 months	Height-for-age z-scores over time
Part B Primary Outcome Measure: Change in weight-for-age z-scores	Baseline to 12 months	Change in weight-for-age z-scores from baseline
Part B Primary Outcome Measure: Change in height-for-age z-scores	Baseline to 12 months	Change in height-for-age z-scores from baseline

Trial Locations

Locations (35)

The Children's Hospital Alabama, University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Stanford University Medical Center; 🇺🇸 Palo Alto, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Nemours Children's Clinic; 🇺🇸 Jacksonville, Florida, United States

University of Miami; 🇺🇸 Miami, Florida, United States

The Nemours Children's Clinic - Orlando; 🇺🇸 Orlando, Florida, United States

Riley Hospital for Children; 🇺🇸 Indianapolis, Indiana, United States

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

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