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

Sponsor
Sonya Heltshe (Other)
Overall Status
Active, not recruiting
CT.gov ID
NCT04509050
Collaborator
Cystic Fibrosis Foundation (Other), University of Washington (Other), University of Alabama at Birmingham (Other)
210
Enrollment
34
Locations
58.4
Anticipated Duration (Months)
6.2
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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 Phase
  • 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.

Study Design

Study Type:
Observational
Anticipated 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 :
Nov 18, 2020
Anticipated Primary Completion Date :
Oct 1, 2025
Anticipated Study Completion Date :
Oct 1, 2025

Arms and Interventions

Arm 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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Part A Primary Outcome Measure: Change in weight-for-age z-scores [Baseline to 12 months]

      Weight-for-age z-scores over time

    2. Part A Primary Outcome Measure: Change in height-for-age z-scores [Baseline to 12 months]

      Height-for-age z-scores over time

    3. Part B Primary Outcome Measure: Change in weight-for-age z-scores [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 [1, 3, 6, 12, and 24 months]

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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 5 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 The Children's Hospital Alabama, University of Alabama at Birmingham Birmingham Alabama United States 35233
    2 Children's Hospital Colorado Aurora Colorado United States 80045
    3 Nemours Children's Clinic Jacksonville Florida United States 32207
    4 University of Miami Miami Florida United States 33136
    5 The Nemours Children's Clinic - Orlando Orlando Florida United States 32827
    6 Riley Hospital for Children Indianapolis Indiana United States 46202
    7 University of Iowa Iowa City Iowa United States 52242
    8 University of Kansas Medical Center Kansas City Kansas United States 66160
    9 Boston Children's Hospital Boston Massachusetts United States 02115
    10 University of Michigan, Michigan Medicine Ann Arbor Michigan United States 48109
    11 Helen DeVos Children's Hospital Grand Rapids Michigan United States 49503
    12 Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota United States 55404
    13 The Minnesota Cystic Fibrosis Center Minneapolis Minnesota United States 55455
    14 Children's Mercy Kansas City Kansas City Missouri United States 64108
    15 St. Louis Children's Hospital Saint Louis Missouri United States 63110
    16 The Cystic Fibrosis Center of Western New York Buffalo New York United States 14203
    17 Children's Hospital of New York New York New York United States 10032
    18 SUNY Upstate Medical University Syracuse New York United States 13210
    19 New York Medical College at Westchester Medical Center Valhalla New York United States 10595
    20 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    21 Nationwide Children's Hospital Columbus Ohio United States 43205
    22 Oklahoma Cystic Fibrosis Center Oklahoma City Oklahoma United States 73104
    23 Oregon Health Sciences University Portland Oregon United States 97239
    24 Hershey Medical Center Pennsylvania State University Hershey Pennsylvania United States 17033
    25 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    26 Children's Hospital of Pittsburgh of UPMC Pittsburgh Pennsylvania United States 15224
    27 University of Texas Southwestern / Children's Health Dallas Texas United States 75247
    28 Cook Children's Medical Center Fort Worth Texas United States 76104
    29 Baylor College of Medicine Houston Texas United States 77030
    30 Primary Children's Cystic Fibrosis Center Salt Lake City Utah United States 84113
    31 Vermont Children's Hospital Burlington Vermont United States 05401
    32 University of Virginia Charlottesville Virginia United States 22903
    33 Seattle Children's Hospital Seattle Washington United States 98105
    34 University of Wisconsin Madison Wisconsin United States 53792

    Sponsors and Collaborators

    • Sonya Heltshe
    • 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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    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:
    Aug 11, 2020
    Last Update Posted:
    Feb 3, 2022
    Last Verified:
    Feb 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Sonya Heltshe, Associate Professor University of Washington, Seattle Children's Hospital
    Cystic Fibrosis
    CF
    CFTR Modulator
    triple combination therapy
    elexacaftor
    tezacaftor
    ivacaftor
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor
    Elexacaftor

    Study Results

    No Results Posted as of Feb 3, 2022