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History of Changes for Study: NCT04509050
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children (BEGIN)
Latest version (submitted February 2, 2022) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 August 10, 2020 None (earliest Version on record)
2 September 8, 2020 Study Status and Study Identification
3 October 30, 2020 Recruitment Status, Study Status, Contacts/Locations and Oversight
4 November 23, 2020 Study Status and Contacts/Locations
5 January 8, 2021 Study Status and Contacts/Locations
6 February 3, 2021 Study Status and Contacts/Locations
7 March 9, 2021 Study Status and Contacts/Locations
8 May 6, 2021 Contacts/Locations and Study Status
9 June 2, 2021 Study Status and Contacts/Locations
10 July 7, 2021 Contacts/Locations and Study Status
11 December 16, 2021 Contacts/Locations and Study Status
12 February 2, 2022 Recruitment Status, Study Status and Contacts/Locations
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Study NCT04509050
Submitted Date:  August 10, 2020 (v1)

Open or close this module Study Identification
Unique Protocol ID: BEGIN-IP-19
Brief Title: Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children (BEGIN)
Official Title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
Secondary IDs:
Open or close this module Study Status
Record Verification: August 2020
Overall Status: Not yet recruiting
Study Start: October 1, 2020
Primary Completion: June 30, 2024 [Anticipated]
Study Completion: June 30, 2024 [Anticipated]
First Submitted: August 6, 2020
First Submitted that
Met QC Criteria:
August 10, 2020
First Posted: August 11, 2020 [Actual]
Last Update Submitted that
Met QC Criteria:
August 10, 2020
Last Update Posted: August 11, 2020 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Sonya Heltshe
Responsible Party: Sponsor-Investigator
Investigator: Sonya Heltshe
Official Title: Associate Professor University of Washington
Affiliation: Seattle Children's Hospital
Collaborators: Cystic Fibrosis Foundation
University of Washington
University of Alabama at Birmingham
Open or close this module Oversight
U.S. FDA-regulated Drug: No
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module 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).
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.

Open or close this module Conditions
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
CF
CFTR Modulator
triple combination therapy
elexacaftor
tezacaftor
ivacaftor
Open or close this module Study Design
Study Type: Observational
Observational Study Model: Cohort
Time Perspective: Prospective
Biospecimen Retention: Samples With DNA
Biospecimen Description: Serum, Plasma, Buffy Coat, Urine, Stool, oropharyngeal (OP) swab, Bronchoalveolar lavage (BAL)
Enrollment: 210 [Anticipated]
Number of Groups/Cohorts 2
Open or close this module Groups and Interventions
Groups/Cohorts Interventions
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
Open or close this module Outcome Measures
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
Open or close this module Eligibility
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

Sampling Method: Non-Probability Sample
Minimum Age:
Maximum Age: 5 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
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.

Open or close this module Contacts/Locations
Central Contact Person: Rachael Buckingham
Telephone: 206-884-7517
Email: rachael.buckingham@seattlechildrens.org
Central Contact Backup: Nicole Rogers
Telephone: 206-884-7554
Email: nicole.rogers@seattlechildrens.org
Study Officials: Bonnie Ramsey, MD
Principal Investigator
Seattle Children's
Lucas Hoffman, MD PhD
Principal Investigator
University of Washington/Seattle Children's
Michael Stalvey, MD
Principal Investigator
University of Alabama at Birmingham
Locations: United States, Alabama
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Contact:Contact: Heather Y Hathorne hhathorne@peds.uab.edu
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Contact:Contact: Meg Anthony Meg.Anthony@childrenscolorado.org
United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
Contact:Contact: Elizabeth DeLuca Elizabeth.DeLuca@nemours.org
University of Miami
Miami, Florida, United States, 33136
Contact:Contact: Alejandra Weisman a.weisman1@miami.edu
The Nemours Children's Clinic - Orlando
Orlando, Florida, United States, 32827
Contact:Contact: Amanda Darling amanda.darling@nemours.org
United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Contact:Contact: Lisa Bendy lbendy@iupui.edu
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
Contact:Contact: Mary Teresi mary-teresi@uiowa.edu
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Contact:Contact: Lawrence Scott lscott2@kumc.edu
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Contact:Contact: Robert Fowler Robert.fowler@childrens.harvard.edu
United States, Michigan
University of Michigan, Michigan Medicine
Ann Arbor, Michigan, United States, 48109
Contact:Contact: Dawn Kruse dmkruse@med.umich.edu
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Contact:Contact: Cynthia M Gile Cynthia.Gile@spectrumhealth.org
United States, Minnesota
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States, 55404
Contact:Contact: Alison Kloste alison.kloste@childrensmn.org
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, United States, 55455
Contact:Contact: CF Trials Contact cftrials@umn.edu
United States, Missouri
Children's Mercy Kansas City
Kansas City, Missouri, United States, 64108
Contact:Contact: Candy Schmoll cschmoll@cmh.edu
St. Louis Children's Hospital
Saint Louis, Missouri, United States, 63110
Contact:Contact: Tina Hicks hickst@wustl.edu
United States, New York
The Cystic Fibrosis Center of Western New York
Buffalo, New York, United States, 14203
Contact:Contact: Nadine Caci ncaci@upa.chob.edu
Children's Hospital of New York
New York, New York, United States, 10032
Contact:Contact: Carmen Liriano crl58@cumc.columbia.edu
SUNY Upstate Medical University
Syracuse, New York, United States, 13210
Contact:Contact: Valoree Suttmore suttmorv@upstate.edu
New York Medical College at Westchester Medical Center
Valhalla, New York, United States, 10595
Contact:Contact: Zachary Messer Zachary_Messer@nymc.edu
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Contact:Contact: Kelly Thornton Kelly.Thornton@cchmc.org
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Contact:Contact: Diana Gilmore Diana.Gilmore@nationwidechildrens.org
United States, Oklahoma
Oklahoma Cystic Fibrosis Center
Oklahoma City, Oklahoma, United States, 73104
Contact:Contact: CF Center Participant Contact cfresearchteam@ouhsc.edu
United States, Oregon
Oregon Health Sciences University
Portland, Oregon, United States, 97239
Contact:Contact: Brendan Klein kleinb@ohsu.edu
United States, Pennsylvania
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, United States, 17033
Contact:Contact: Diane M Kitch dkitch@pennstatehealth.psu.edu
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Contact:Contact: Christina Kubrak kubrak@email.chop.edu
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Contact:Contact: Elizabeth Hartigan elizabeth.hartigan@chp.edu
United States, Texas
University of Texas Southwestern / Children's Health
Dallas, Texas, United States, 75247
Contact:Contact: Daniyal Kamal Daniyal.Kamal@childrens.com
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Contact:Contact: Heather Urbanek Heather.Urbanek@cookchildrens.org
Baylor College of Medicine
Houston, Texas, United States, 77030
Contact:Contact: Nicoline Schaap nxschaap@texaschildrens.org
United States, Utah
Primary Children's Cystic Fibrosis Center
Salt Lake City, Utah, United States, 84113
Contact:Contact: Jane B Vroom jane.vroom@hsc.utah.edu
United States, Vermont
Vermont Children's Hospital
Burlington, Vermont, United States, 05401
Contact:Contact: Julie Sweet julie.sweet@uvmhealth.org
United States, Virginia
University of Virginia
Charlottesville, Virginia, United States, 22903
Contact:Contact: Christie Aderholt cla6e@virginia.edu
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Contact:Contact: Sharon McNamara sharon.mcnamara@seattlechildrens.org
United States, Wisconsin
University of Wisconsin
Madison, Wisconsin, United States, 53792
Contact:Contact: Melanie Nelson mnelson@pediatrics.wisc.edu
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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