A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
Status: Recruiting
Location: See all (35) 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: 10
Healthy Volunteers: f
View:
• 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.
Locations
United States
Alabama
The Children's Hospital Alabama, University of Alabama at Birmingham
RECRUITING
Birmingham
California
Stanford University Medical Center
RECRUITING
Palo Alto
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Florida
Nemours Children's Clinic
RECRUITING
Jacksonville
University of Miami
RECRUITING
Miami
The Nemours Children's Clinic - Orlando
RECRUITING
Orlando
Iowa
University of Iowa
RECRUITING
Iowa City
Indiana
Riley Hospital for Children
RECRUITING
Indianapolis
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Michigan
University of Michigan, Michigan Medicine
RECRUITING
Ann Arbor
Helen DeVos Children's Hospital
RECRUITING
Grand Rapids
Minnesota
Children's Hospitals and Clinics of Minnesota
RECRUITING
Minneapolis
The Minnesota Cystic Fibrosis Center
RECRUITING
Minneapolis
Missouri
Children's Mercy Kansas City
RECRUITING
Kansas City
St. Louis Children's Hospital
RECRUITING
St Louis
New York
The Cystic Fibrosis Center of Western New York
RECRUITING
Buffalo
Children's Hospital of New York
RECRUITING
New York
SUNY Upstate Medical University
RECRUITING
Syracuse
New York Medical College at Westchester Medical Center
RECRUITING
Valhalla
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Nationwide Children's Hospital
RECRUITING
Columbus
Oklahoma
Oklahoma Cystic Fibrosis Center
RECRUITING
Oklahoma City
Oregon
Oregon Health Sciences University
RECRUITING
Portland
Pennsylvania
Hershey Medical Center Pennsylvania State University
RECRUITING
Hershey
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Children's Hospital of Pittsburgh of UPMC
RECRUITING
Pittsburgh
Texas
University of Texas Southwestern / Children's Health
RECRUITING
Dallas
Cook Children's Medical Center
RECRUITING
Fort Worth
Baylor College of Medicine
RECRUITING
Houston
Utah
Primary Children's Cystic Fibrosis Center
RECRUITING
Salt Lake City
Virginia
University of Virginia
RECRUITING
Charlottesville
Vermont
Vermont Children's Hospital
RECRUITING
Burlington
Washington
Seattle Children's Hospital
RECRUITING
Seattle
Wisconsin
University of Wisconsin
RECRUITING
Madison
Contact Information
Primary
Rachael Buckingham
rachael.buckingham@seattlechildrens.org
206-884-7517
Backup
Anna Mead
anna.mead@seattlechildrens.org
206-884-7531
Time Frame
Start Date: 2020-11-18
Estimated Completion Date: 2029-12-01
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.
Authors
Related Therapeutic Areas
Sponsors
Leads: Sonya Heltshe
Collaborators: University of Washington, Cystic Fibrosis Foundation, University of Alabama at Birmingham