RecruitingNCT04509050

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

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

Sponsor

Sonya Heltshe

Enrollment

210 participants

Start Date

Nov 18, 2020

Study Type

OBSERVATIONAL

Conditions

Cystic Fibrosis

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).

Eligibility

Max Age: 10 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Ivacaftor or elexacaftor/tezacaftor/ivacaftor for people with cystic fibrosis. The study is currently recruiting participants at 35 locations. People eligible for this study include up to age 10 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGIvacaftor 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

Locations(35)

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

Birmingham, Alabama, United States

Stanford University Medical Center

Palo Alto, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours Children's Clinic

Jacksonville, Florida, United States

University of Miami

Miami, Florida, United States

The Nemours Children's Clinic - Orlando

Orlando, Florida, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

University of Iowa

Iowa City, Iowa, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Boston Children's Hospital

Boston, Massachusetts, United States

University of Michigan, Michigan Medicine

Ann Arbor, Michigan, United States

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, United States

The Minnesota Cystic Fibrosis Center

Minneapolis, Minnesota, United States

Children's Mercy Kansas City

Kansas City, Missouri, United States

St. Louis Children's Hospital

St Louis, Missouri, United States

The Cystic Fibrosis Center of Western New York

Buffalo, New York, United States

Children's Hospital of New York

New York, New York, United States

SUNY Upstate Medical University

Syracuse, New York, United States

New York Medical College at Westchester Medical Center

Valhalla, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Oklahoma Cystic Fibrosis Center

Oklahoma City, Oklahoma, United States

Oregon Health Sciences University

Portland, Oregon, United States

Hershey Medical Center Pennsylvania State University

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

University of Texas Southwestern / Children's Health

Dallas, Texas, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

Primary Children's Cystic Fibrosis Center

Salt Lake City, Utah, United States

Vermont Children's Hospital

Burlington, Vermont, United States

University of Virginia

Charlottesville, Virginia, United States

Seattle Children's Hospital

Seattle, Washington, United States

University of Wisconsin

Madison, Wisconsin, United States

View Full Details on ClinicalTrials.gov

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NCT04509050

Related Trials

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

Conditions

Summary

Eligibility

Plain Language Summary

Interventions

Locations(35)

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