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

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ClinicalTrials.gov Identifier: NCT04509050
Recruitment Status : Recruiting
First Posted : August 11, 2020
Last Update Posted : June 4, 2021
Sponsor:
Collaborators:
Cystic Fibrosis Foundation
University of Washington
University of Alabama at Birmingham
Information provided by (Responsible Party):
Sonya Heltshe, Seattle Children's Hospital

Brief 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).

Condition or disease Intervention/treatment
Cystic Fibrosis Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor

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.

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Study Type : Observational
Estimated Enrollment : 210 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
Actual Study Start Date : November 18, 2020
Estimated Primary Completion Date : October 1, 2025
Estimated Study Completion Date : October 1, 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

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




Primary Outcome Measures :
  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: 1, 3, 6, 12, and 24 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: 1, 3, 6, 12, and 24 months ]
    Change in height-for-age z-scores from baseline


Biospecimen Retention:   Samples With DNA
Serum, Plasma, Buffy Coat, Urine, Stool, oropharyngeal (OP) swab, Bronchoalveolar lavage (BAL)


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
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

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04509050


Contacts
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Contact: Rachael Buckingham 206-884-7517 rachael.buckingham@seattlechildrens.org
Contact: Nicole Rogers 206-884-7554 nicole.rogers@seattlechildrens.org

Locations
Show Show 34 study locations
Sponsors and Collaborators
Sonya Heltshe
Cystic Fibrosis Foundation
University of Washington
University of Alabama at Birmingham
Investigators
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Principal Investigator: Bonnie Ramsey, MD Seattle Children's
Principal Investigator: Lucas Hoffman, MD PhD University of Washington/Seattle Children's
Principal Investigator: Michael Stalvey, MD University of Alabama at Birmingham
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Responsible Party: Sonya Heltshe, Associate Professor University of Washington, Seattle Children's Hospital
ClinicalTrials.gov Identifier: NCT04509050    
Other Study ID Numbers: BEGIN-OB-19
First Posted: August 11, 2020    Key Record Dates
Last Update Posted: June 4, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sonya Heltshe, Seattle Children's Hospital:
Cystic Fibrosis
CF
CFTR Modulator
triple combination therapy
elexacaftor
tezacaftor
ivacaftor
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action