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

May 4, 2023 updated by: Sonya Heltshe

A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN 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 on children with cystic fibrosis (CF).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Anticipated)

210

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Recruiting
        • The Children's Hospital Alabama, University of Alabama at Birmingham
        • Contact:
    • Colorado
    • Florida
      • Jacksonville, Florida, United States, 32207
        • Active, not recruiting
        • Nemours Children's Clinic
      • Miami, Florida, United States, 33136
      • Orlando, Florida, United States, 32827
        • Active, not recruiting
        • The Nemours Children's Clinic - Orlando
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Riley Hospital for Children
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
    • Massachusetts
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Active, not recruiting
        • University of Michigan, Michigan Medicine
      • Grand Rapids, Michigan, United States, 49503
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • The Minnesota Cystic Fibrosis Center
        • Contact:
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Children's Mercy Kansas City
        • Contact:
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • St. Louis Children's Hospital
        • Contact:
    • New York
      • Buffalo, New York, United States, 14203
        • Active, not recruiting
        • The Cystic Fibrosis Center of Western New York
      • New York, New York, United States, 10032
      • Syracuse, New York, United States, 13210
      • Valhalla, New York, United States, 10595
        • Active, not recruiting
        • New York Medical College at Westchester Medical Center
    • Ohio
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Active, not recruiting
        • Oklahoma Cystic Fibrosis Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health Sciences University
        • Contact:
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Active, not recruiting
        • Hershey Medical Center Pennsylvania State University
      • Philadelphia, Pennsylvania, United States, 19104
      • Pittsburgh, Pennsylvania, United States, 15224
    • Texas
      • Dallas, Texas, United States, 75247
      • Fort Worth, Texas, United States, 76104
        • Active, not recruiting
        • Cook Children's Medical Center
      • Houston, Texas, United States, 77030
    • Utah
      • Salt Lake City, Utah, United States, 84113
    • Vermont
      • Burlington, Vermont, United States, 05401
        • Active, not recruiting
        • Vermont Children's Hospital
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Active, not recruiting
        • University of Virginia
    • Washington
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Active, not recruiting
        • University of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 5 years (Child)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Part A - children with confirmed diagnosis of cystic fibrosis who are less than 5 years of age and not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy

Part B - children with confirmed diagnosis of cystic fibrosis who are less than 6 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Part A
Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
Part B
Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the Part A cohort of this study may enroll into the Part B cohort if they become eligible for these CFTR modulator therapies and plan to start them.
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
Other Names:
  • Kalydeco or Trikafta
  • Vertex (VX)-770 or VX-445/VX-661/VX-770

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline to 12 months
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
Baseline to 12 months
Part B Primary Outcome Measure: Change in weight-for-age z-scores
Time Frame: 1, 3, 6, 12, and 24 months
Change in weight-for-age z-scores from baseline
1, 3, 6, 12, and 24 months
Part B Primary Outcome Measure: Change in height-for-age z-scores
Time Frame: 1, 3, 6, 12, and 24 months
Change in height-for-age z-scores from baseline
1, 3, 6, 12, and 24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sonya Heltshe

Collaborators

University of Alabama at Birmingham
University of Washington
Cystic Fibrosis Foundation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 18, 2020

Primary Completion (Anticipated)

October 1, 2025

Study Completion (Anticipated)

October 1, 2025

Study Registration Dates

First Submitted

August 6, 2020

First Submitted That Met QC Criteria

August 10, 2020

First Posted (Actual)

August 11, 2020

Study Record Updates

Last Update Posted (Estimate)

May 5, 2023

Last Update Submitted That Met QC Criteria

May 4, 2023

Last Verified

May 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BEGIN-OB-19

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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