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

Status: Active, not recruiting
Location: See all (34) locations...
Intervention Type: Drug
Study Type: Observational
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
Participation Requirements
Sex: All
Maximum Age: 5
Healthy Volunteers: No
View:

• 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.

Locations
United States
Alabama
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham
Colorado
Children's Hospital Colorado
Aurora
Florida
Nemours Children's Clinic
Jacksonville
University of Miami
Miami
The Nemours Children's Clinic - Orlando
Orlando
Iowa
University of Iowa
Iowa City
Indiana
Riley Hospital for Children
Indianapolis
Kansas
University of Kansas Medical Center
Kansas City
Massachusetts
Boston Children's Hospital
Boston
Michigan
University of Michigan, Michigan Medicine
Ann Arbor
Helen DeVos Children's Hospital
Grand Rapids
Minnesota
Children's Hospitals and Clinics of Minnesota
Minneapolis
The Minnesota Cystic Fibrosis Center
Minneapolis
Missouri
Children's Mercy Kansas City
Kansas City
St. Louis Children's Hospital
Saint Louis
New York
The Cystic Fibrosis Center of Western New York
Buffalo
Children's Hospital of New York
New York
SUNY Upstate Medical University
Syracuse
New York Medical College at Westchester Medical Center
Valhalla
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Nationwide Children's Hospital
Columbus
Oklahoma
Oklahoma Cystic Fibrosis Center
Oklahoma City
Oregon
Oregon Health Sciences University
Portland
Pennsylvania
Hershey Medical Center Pennsylvania State University
Hershey
Children's Hospital of Philadelphia
Philadelphia
Children's Hospital of Pittsburgh of UPMC
Pittsburgh
Texas
University of Texas Southwestern / Children's Health
Dallas
Cook Children's Medical Center
Fort Worth
Baylor College of Medicine
Houston
Utah
Primary Children's Cystic Fibrosis Center
Salt Lake City
Virginia
University of Virginia
Charlottesville
Vermont
Vermont Children's Hospital
Burlington
Washington
Seattle Children's Hospital
Seattle
Wisconsin
University of Wisconsin
Madison
Time Frame
Start Date: November 18, 2020
Estimated Completion Date: October 1, 2025
Participants
Target number of participants: 210
Treatments
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.
Related Therapeutic Areas
Sponsors
Collaborators: Cystic Fibrosis Foundation, University of Alabama at Birmingham, University of Washington
Leads: Sonya Heltshe

This content was sourced from clinicaltrials.gov

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