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).
Conditions
Cystic Fibrosis
Recruitment Status
Active, not recruiting
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 6 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 6 visits over a period of up to 3 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 one "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and five "after ivacaftor or elex/tez/iva" visits over a 24-month follow-up period.
Eligibility Criteria
Inclusion Criteria:
Part A:
Less than 5 years of age at the first study visit.
Documentation of a CF diagnosis.
Part B:
Participated in Part A OR less than 6 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 180 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.
Intervention
Intervention Type
Intervention Name
Drug
Ivacaftor or elexacaftor/tezacaftor/ivacaftor
Gender
All
Min Age
N/A
Max Age
5 Years
Download Date
February 3, 2022
Principal Investigator
Sonya Heltshe
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: