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

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).

Conditions

Cystic Fibrosis

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Study Overview

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Study Details

Study overview

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).

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

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

Condition
Cystic Fibrosis
Intervention / Treatment

-

Contacts and Locations

Birmingham

The Children's Hospital Alabama, University of Alabama at Birmingham, Birmingham, Alabama, United States, 35233

Palo Alto

Stanford University Medical Center, Palo Alto, California, United States, 94304

Aurora

Children's Hospital Colorado, Aurora, Colorado, United States, 80045

Jacksonville

Nemours Children's Clinic, Jacksonville, Florida, United States, 32207

Miami

University of Miami, Miami, Florida, United States, 33136

Orlando

The Nemours Children's Clinic - Orlando, Orlando, Florida, United States, 32827

Indianapolis

Riley Hospital for Children, Indianapolis, Indiana, United States, 46202

Iowa City

University of Iowa, Iowa City, Iowa, United States, 52242

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

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

Ages Eligible for Study

to 10 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

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

2029-12-01