Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
NCT ID: NCT04509050
Last Updated: 2024-08-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
210 participants
OBSERVATIONAL
2020-11-18
2029-12-01
Brief Summary
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Detailed Description
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Total duration of the study is expected to be 10 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth factors in early childhood and assess the feasibility of additional measurements. In Part A, subjects will have up to 8 visits over a period of up to 5 years.
Part B will be a prospective longitudinal study to observe the effects of administration of either ivacaftor or elexacaftor/tezacaftor/ivacaftor (elex/tez/iva) on growth. In Part B, subjects will have 1 "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and 8 "after ivacaftor or elex/tez/iva" visits over a 60-month follow-up period.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Part A
Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
No interventions assigned to this group
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.
Ivacaftor 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
Interventions
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Ivacaftor 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
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
10 Years
ALL
No
Sponsors
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Cystic Fibrosis Foundation
OTHER
University of Washington
OTHER
University of Alabama at Birmingham
OTHER
Sonya Heltshe
OTHER
Responsible Party
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Sonya Heltshe
Associate Professor University of Washington
Principal Investigators
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Bonnie Ramsey, MD
Role: PRINCIPAL_INVESTIGATOR
Seattle Children's Hospital
Lucas Hoffman, MD PhD
Role: PRINCIPAL_INVESTIGATOR
University of Washington/Seattle Children's
Katie Larson Ode, MD
Role: PRINCIPAL_INVESTIGATOR
University of Iowa
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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BEGIN-OB-19
Identifier Type: -
Identifier Source: org_study_id
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