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

Purpose

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).

Condition

  • Cystic Fibrosis

Eligibility

Eligible Ages
Under 10 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
Part A Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
Part B 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.
  • Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor
    In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators
    Other names:
    • Kalydeco or Trikafta
    • Vertex (VX)-770 or VX-445/VX-661/VX-770

Recruiting Locations

The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama 35233
Contact:
Heather Hathorne
hyhathorne@uabmc.edu

More Details

Status
Recruiting
Sponsor
Sonya Heltshe

Study Contact

Rachael Buckingham
206-884-7517
rachael.buckingham@seattlechildrens.org

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.