RECRUITING

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

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Conditions

Cystic FibrosisCFCFTR Modulatortriple combination therapyelexacaftortezacaftorivacaftor

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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 on children with cystic fibrosis (CF).

Official Title

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

Quick Facts

Study Start:2020-11-18
Study Completion:2029-12-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04509050

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 10 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Part A:
  2. * Less than 10 years of age at the first study visit.
  3. * Documentation of a CF diagnosis.
  4. * Participated in Part A OR less than 7 years of age at the first study visit.
  5. * Documentation of a CF diagnosis.
  6. * CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor).
  7. * Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.
  1. * Part A and Part B:
  2. * Use of an investigational drug within 28 days prior to and including the first study visit.
  3. * Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit.
  4. * Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.

Contacts and Locations

Study Contact

Rachael Buckingham
CONTACT
206-884-7517
rachael.buckingham@seattlechildrens.org
Anna Mead
CONTACT
206-884-7531
anna.mead@seattlechildrens.org

Principal Investigator

Bonnie Ramsey, MD
PRINCIPAL_INVESTIGATOR
Seattle Children's
Lucas Hoffman, MD PhD
PRINCIPAL_INVESTIGATOR
University of Washington/Seattle Children's
Katie Larson Ode, MD
PRINCIPAL_INVESTIGATOR
University of Iowa

Study Locations (Sites)

The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, 35233
United States
Stanford University Medical Center
Palo Alto, California, 94304
United States
Children's Hospital Colorado
Aurora, Colorado, 80045
United States
Nemours Children's Clinic
Jacksonville, Florida, 32207
United States
University of Miami
Miami, Florida, 33136
United States
The Nemours Children's Clinic - Orlando
Orlando, Florida, 32827
United States
Riley Hospital for Children
Indianapolis, Indiana, 46202
United States
University of Iowa
Iowa City, Iowa, 52242
United States
University of Kansas Medical Center
Kansas City, Kansas, 66160
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
University of Michigan, Michigan Medicine
Ann Arbor, Michigan, 48109
United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
United States
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, 55455
United States
Children's Mercy Kansas City
Kansas City, Missouri, 64108
United States
St. Louis Children's Hospital
Saint Louis, Missouri, 63110
United States
The Cystic Fibrosis Center of Western New York
Buffalo, New York, 14203
United States
Children's Hospital of New York
New York, New York, 10032
United States
SUNY Upstate Medical University
Syracuse, New York, 13210
United States
New York Medical College at Westchester Medical Center
Valhalla, New York, 10595
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
Oklahoma Cystic Fibrosis Center
Oklahoma City, Oklahoma, 73104
United States
Oregon Health Sciences University
Portland, Oregon, 97239
United States
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, 17033
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
United States
University of Texas Southwestern / Children's Health
Dallas, Texas, 75207
United States
Cook Children's Medical Center
Fort Worth, Texas, 76104
United States
Baylor College of Medicine
Houston, Texas, 77030
United States
Primary Children's Cystic Fibrosis Center
Salt Lake City, Utah, 84113
United States
Vermont Children's Hospital
Burlington, Vermont, 05401
United States
University of Virginia
Charlottesville, Virginia, 22903
United States
Seattle Children's Hospital
Seattle, Washington, 98105
United States
University of Wisconsin
Madison, Wisconsin, 53792
United States

Collaborators and Investigators

Sponsor: Sonya Heltshe

  • Bonnie Ramsey, MD, PRINCIPAL_INVESTIGATOR, Seattle Children's
  • Lucas Hoffman, MD PhD, PRINCIPAL_INVESTIGATOR, University of Washington/Seattle Children's
  • Katie Larson Ode, MD, PRINCIPAL_INVESTIGATOR, University of Iowa

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-11-18
Study Completion Date2029-12-01

Study Record Updates

Study Start Date2020-11-18
Study Completion Date2029-12-01

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

  • Cystic Fibrosis