Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, CF, CFTR Modulator, triple combination therapy, elexacaftor, tezacaftor, ivacaftor
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.
Interventions
In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators
Sponsors
Sonya Heltshe
Cystic Fibrosis Foundation
University of Washington
University of Alabama at Birmingham
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 10 Years
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Contacts
CONTACTRachael Buckingham
CONTACTAnna Mead
PRINCIPAL_INVESTIGATORBonnie Ramsey, MD
Seattle Children's Hospital
PRINCIPAL_INVESTIGATORLucas Hoffman, MD PhD
University of Washington/Seattle Children's
PRINCIPAL_INVESTIGATORKatie Larson Ode, MD
University of Iowa
Outcome results
None listed