Recruiting

CFTR Function

Sponsor:

Sonya Heltshe

Code:

NCT04509050

Conditions

Cystic Fibrosis

Eligibility Criteria

Sex: All

Age: 0 - 10

Healthy Volunteers: Not accepted

Interventions

Ivacaftor or elexacaftor/tezacaftor/ivacaftor

Study Details

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).

Conditions

Cystic Fibrosis

Study ID

NCT04509050

Start date

Nov 18, 2020

Status verified date

Jul, 2026

Completion date

Dec 31, 2027

Anticipated

Primary completion date

Dec 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 10

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Part A:

  • Less than 10 years of age at the first study visit.
  • Documentation of a CF diagnosis.

Part B:

  • Participated in Part A OR less than 7 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 28 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 Design

Enrollment

210 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Interventions

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

Primary outcome measure

  • Part A Primary Outcome Measure: Change in weight-for-age z-scores [ Time Frame: Baseline to 12 months ]
  • Part A Primary Outcome Measure: Change in height-for-age z-scores [ Time Frame: Baseline to 12 months ]
  • Part B Primary Outcome Measure: Change in weight-for-age z-scores [ Time Frame: Baseline to 12 months ]
  • Part B Primary Outcome Measure: Change in height-for-age z-scores [ Time Frame: Baseline to 12 months ]

Central Contacts and Locations

Central contacts

Rachael Buckingham

206-884-7517rachael.buckingham@seattlechildrens.org

Anna Mead

206-884-7531anna.mead@seattlechildrens.org

Locations

The Children's Hospital Alabama, University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Heather Hathorne

hyhathorne@uabmc.edu

Stanford University Medical Center

Recruiting

Palo Alto, California, United States, 94304

Contacts

Jacquelyn Spano

jmzirbes@stanford.edu

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Mary Cross

mary.cross@childrenscolorado.org

Nemours Children's Clinic

Recruiting

Jacksonville, Florida, United States, 32207

Contacts

Jennifer (Jenn) Gafford

Jennifer.gafford@nemours.org

University of Miami

Recruiting

Miami, Florida, United States, 33136

Contacts

Ylber (Ivan) Whitaker

yiw2@miami.edu

The Nemours Children's Clinic - Orlando

Recruiting

Orlando, Florida, United States, 32827

Contacts

Sherry Wooldridge

sherry.wooldridge@nemours.org

Riley Hospital for Children

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Lisa Bendy

lbendy@iu.edu

University of Iowa

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Mary Teresi

mary-teresi@uiowa.edu

University of Kansas Medical Center

Recruiting

Kansas City, Kansas, United States, 66160

Contacts

Lawrence Scott

lscott2@kumc.edu

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Robert Fowler

Robert.fowler@childrens.harvard.edu

University of Michigan, Michigan Medicine

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Dawn Kruse

dmkruse@med.umich.edu

Helen DeVos Children's Hospital

Recruiting

Grand Rapids, Michigan, United States, 49503

Contacts

Andrew James

andrew.james@corewellhealth.org

Children's Hospitals and Clinics of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55404

Contacts

Camerone Bey

Camerone.bey@childrensmn.org

The Minnesota Cystic Fibrosis Center

Recruiting

Minneapolis, Minnesota, United States, 55455

Contacts

CF Trials Contact

cftrials@umn.edu

Children's Mercy Kansas City

Recruiting

Kansas City, Missouri, United States, 64108

Contacts

Candy Schmoll

cschmoll@cmh.edu

St. Louis Children's Hospital

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Irma Bauer

irmabauer@wustl.edu

The Cystic Fibrosis Center of Western New York

Recruiting

Buffalo, New York, United States, 14203

Contacts

Christine Roach

croach@upa.chob.edu

Children's Hospital of New York

Recruiting

New York, New York, United States, 10032

Contacts

Hossein Sadeghi

HS762@cumc.Columbia.edu

SUNY Upstate Medical University

Recruiting

Syracuse, New York, United States, 13210

Contacts

Mary Forell

forellm@upstate.edu

New York Medical College at Westchester Medical Center

Recruiting

Valhalla, New York, United States, 10595

Contacts

Zachary Messer

Zachary_Messer@nymc.edu

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Kelly Thornton

Kelly.Thornton@cchmc.org

Nationwide Children's Hospital

Recruiting

Columbus, Ohio, United States, 43205

Contacts

Diana Gilmore

Diana.Gilmore@nationwidechildrens.org

Oklahoma Cystic Fibrosis Center

Recruiting

Oklahoma City, Oklahoma, United States, 73104

Contacts

CF Center Participant Contact

cfresearchteam@ouhsc.edu

Oregon Health Sciences University

Recruiting

Portland, Oregon, United States, 97239

Contacts

Jenna Bucher

bucherj@ohsu.edu

Hershey Medical Center Pennsylvania State University

Recruiting

Hershey, Pennsylvania, United States, 17033

Contacts

Diane M Kitch

dkitch@pennstatehealth.psu.edu

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Erin Donnelly

Donnellye4@email.chop.edu

Children's Hospital of Pittsburgh of UPMC

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contacts

Elizabeth Hartigan

elizabeth.hartigan@chp.edu

University of Texas Southwestern / Children's Health

Recruiting

Dallas, Texas, United States, 75207

Contacts

Keianna Brown

Keianna.brown@utsouthwestern.edu

Cook Children's Medical Center

Recruiting

Fort Worth, Texas, United States, 76104

Contacts

Anna Reyes

anna.reyes@cookchildrens.org

Baylor College of Medicine

Recruiting

Houston, Texas, United States, 77030

Contacts

Tracy Mosely

tlmosely@texaschildrens.org

Primary Children's Cystic Fibrosis Center

Recruiting

Salt Lake City, Utah, United States, 84113

Contacts

Jane B Vroom

jane.vroom@hsc.utah.edu

Vermont Children's Hospital

Recruiting

Burlington, Vermont, United States, 05401

Contacts

Julie Sweet

julie.sweet@uvmhealth.org

University of Virginia

Recruiting

Charlottesville, Virginia, United States, 22903

Contacts

Erica Tharrington

SFV6AB@hscmail.mcc.virginia.edu

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Contacts

Sharon McNamara

sharon.mcnamara@seattlechildrens.org

University of Wisconsin

Recruiting

Madison, Wisconsin, United States, 53792

Contacts

Melanie Nelson

mnelson@pediatrics.wisc.edu

More Information

Sponsor

Sonya Heltshe

Last update posted

Jul 8, 2026

Last verified

Jul, 2026

Keywords

  • Cystic Fibrosis
  • CF
  • CFTR Modulator
  • triple combination therapy
  • elexacaftor
  • tezacaftor
  • ivacaftor

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Sonya Heltshe on 2026-07-08.