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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04509050
Recruitment Status : Recruiting
First Posted : August 11, 2020
Last Update Posted : May 6, 2023
Sponsor:
Collaborators:
Cystic Fibrosis Foundation
University of Washington
University of Alabama at Birmingham
Information provided by (Responsible Party):
Sonya Heltshe, Seattle Children's Hospital

Tracking Information
First Submitted Date August 6, 2020
First Posted Date August 11, 2020
Last Update Posted Date May 6, 2023
Actual Study Start Date November 18, 2020
Estimated Primary Completion Date October 1, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 10, 2020)
  • 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
  • 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
  • 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
  • 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
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Official Title A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
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).
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
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Serum, Plasma, Buffy Coat, Urine, Stool, oropharyngeal (OP) swab, Bronchoalveolar lavage (BAL)
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
Condition Cystic 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
Other Names:
  • Kalydeco or Trikafta
  • Vertex (VX)-770 or VX-445/VX-661/VX-770
Study Groups/Cohorts
  • 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.
    Intervention: Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: August 10, 2020)		 210
Original Estimated Enrollment Same as current
Estimated Study Completion Date October 1, 2025
Estimated Primary Completion Date October 1, 2025   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages up to 5 Years   (Child)
Accepts Healthy Volunteers No
Contacts
Contact: Rachael Buckingham 206-884-7517 rachael.buckingham@seattlechildrens.org
Contact: Nicole Rogers 206-884-7554 nicole.rogers@seattlechildrens.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04509050
Other Study ID Numbers BEGIN-OB-19
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Current Responsible Party Sonya Heltshe, Seattle Children's Hospital
Original Responsible Party Same as current
Current Study Sponsor Sonya Heltshe
Original Study Sponsor Same as current
Collaborators
  • Cystic Fibrosis Foundation
  • University of Washington
  • University of Alabama at Birmingham
Investigators
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
PRS Account Seattle Children's Hospital
Verification Date May 2023