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NCT Number: NCT04509050

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

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).

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

Up to 10 year

Sex eligibility

All sexes

Study type

Observational

Primary location

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

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Ivacaftor or elexacaftor/tezacaftor/ivacaftor

Drug

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

Primary outcomes

  1. Part A Primary Outcome Measure: Change in weight-for-age z-scores

    Time frame: Baseline to 12 months

    Weight-for-age z-scores over time

  2. Part A Primary Outcome Measure: Change in height-for-age z-scores

    Time frame: Baseline to 12 months

    Height-for-age z-scores over time

  3. Part B Primary Outcome Measure: Change in weight-for-age z-scores

    Time frame: Baseline to 12 months

    Change in weight-for-age z-scores from baseline

  4. Part B Primary Outcome Measure: Change in height-for-age z-scores

    Time frame: Baseline to 12 months

    Change in height-for-age z-scores from baseline

Study contacts

Contact information is provided by the study sponsor or research team.

Anna Mead

CONTACT

[email protected]

206-884-7531

Rachael Buckingham

CONTACT

[email protected]

206-884-7517

Sponsors and collaborators

Lead sponsor

Sonya Heltshe

Other

Collaborators

  • Cystic Fibrosis Foundation
  • University of Alabama at Birmingham
  • University of Washington

Registry information

Official study title

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

Acronym: BEGIN

Important dates

Study start
2020
Primary completion
2027
Study completion
2027
First posted
Aug 11, 2020
Registry last updated
Jul 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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