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Recruiting NCT04509050

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

— Observational Cystic Fibrosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Who it may be relevant to
Registry conditions: Cystic Fibrosis. Basic parameters: up to 10 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)

Overview

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

  • 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

Primary outcome measures

  • Part A Primary Outcome Measure: Change in weight-for-age z-scores [Time frame: Baseline to 12 months]
  • Part A Primary Outcome Measure: Change in height-for-age z-scores [Time frame: Baseline to 12 months]
  • Part B Primary Outcome Measure: Change in weight-for-age z-scores [Time frame: Baseline to 12 months]
  • Part B Primary Outcome Measure: Change in height-for-age z-scores [Time frame: Baseline to 12 months]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 35 centers
  • The Children's Hospital Alabama, University of Alabama at Birmingham — Birmingham
  • Stanford University Medical Center — Palo Alto
  • Children's Hospital Colorado — Aurora
  • Nemours Children's Clinic — Jacksonville
  • University of Miami — Miami
  • The Nemours Children's Clinic - Orlando — Orlando
  • Riley Hospital for Children — Indianapolis
  • University of Iowa — Iowa City
  • … and 27 more centers

Identifiers

NCT: NCT04509050 · BEGIN-OB-19

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗