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

Cystic Fibrosis Clinical Trial

— BEGIN  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04509050
• Other study ID #: BEGIN-OB-19
• Status: Recruiting
• Start date: November 18, 2020
• Est. completion date: October 1, 2025

Study information

• Verified date: May 2023
• Source: Seattle Children's Hospital
• Contact: Rachael Buckingham
• Phone: 206-884-7517
• Email: rachael.buckingham[@]seattlechildrens.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 210
• Est. completion date: October 1, 2025
• Est. primary completion date: October 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 5 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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

Locations

Country	Name	City	State
United States	University of Michigan, Michigan Medicine	Ann Arbor	Michigan
United States	Children's Hospital Colorado	Aurora	Colorado
United States	The Children's Hospital Alabama, University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	The Cystic Fibrosis Center of Western New York	Buffalo	New York
United States	Vermont Children's Hospital	Burlington	Vermont
United States	University of Virginia	Charlottesville	Virginia
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Texas Southwestern / Children's Health	Dallas	Texas
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Helen DeVos Children's Hospital	Grand Rapids	Michigan
United States	Hershey Medical Center Pennsylvania State University	Hershey	Pennsylvania
United States	Baylor College of Medicine	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	University of Iowa	Iowa City	Iowa
United States	Nemours Children's Clinic	Jacksonville	Florida
United States	Children's Mercy Kansas City	Kansas City	Missouri
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Wisconsin	Madison	Wisconsin
United States	University of Miami	Miami	Florida
United States	Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	The Minnesota Cystic Fibrosis Center	Minneapolis	Minnesota
United States	Children's Hospital of New York	New York	New York
United States	Oklahoma Cystic Fibrosis Center	Oklahoma City	Oklahoma
United States	The Nemours Children's Clinic - Orlando	Orlando	Florida
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Oregon Health Sciences University	Portland	Oregon
United States	St. Louis Children's Hospital	Saint Louis	Missouri
United States	Primary Children's Cystic Fibrosis Center	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	SUNY Upstate Medical University	Syracuse	New York
United States	New York Medical College at Westchester Medical Center	Valhalla	New York

Sponsors (4)

Lead Sponsor	Collaborator
Sonya Heltshe	Cystic Fibrosis Foundation, University of Alabama at Birmingham, University of Washington

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part A Primary Outcome Measure: Change in weight-for-age z-scores	Weight-for-age z-scores over time	Baseline to 12 months
Primary	Part A Primary Outcome Measure: Change in height-for-age z-scores	Height-for-age z-scores over time	Baseline to 12 months
Primary	Part B Primary Outcome Measure: Change in weight-for-age z-scores	Change in weight-for-age z-scores from baseline	1, 3, 6, 12, and 24 months
Primary	Part B Primary Outcome Measure: Change in height-for-age z-scores	Change in height-for-age z-scores from baseline	1, 3, 6, 12, and 24 months