Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
BEGIN
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
1 other identifier
observational
210
1 country
35
Brief 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Nov 2020
Longer than P75 for all trials
35 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 6, 2020
CompletedFirst Posted
Study publicly available on registry
August 11, 2020
CompletedStudy Start
First participant enrolled
November 18, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2029
August 12, 2024
August 1, 2024
9 years
August 6, 2020
August 8, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
Part A Primary Outcome Measure: Change in weight-for-age z-scores
Weight-for-age z-scores over time
Baseline to 12 months
Part A Primary Outcome Measure: Change in height-for-age z-scores
Height-for-age z-scores over time
Baseline to 12 months
Part B Primary Outcome Measure: Change in weight-for-age z-scores
Change in weight-for-age z-scores from baseline
Baseline to 12 months
Part B Primary Outcome Measure: Change in height-for-age z-scores
Change in height-for-age z-scores from baseline
Baseline to 12 months
Study Arms (2)
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.
Interventions
In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators
Eligibility Criteria
Part A - children with confirmed diagnosis of cystic fibrosis who are less than 10 years of age and not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy Part B - children with confirmed diagnosis of cystic fibrosis who are less than 7 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Sonya Heltshelead
- Cystic Fibrosis Foundationcollaborator
- University of Washingtoncollaborator
- University of Alabama at Birminghamcollaborator
Study Sites (35)
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Stanford University Medical Center
Palo Alto, California, 94304, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Nemours Children's Clinic
Jacksonville, Florida, 32207, United States
University of Miami
Miami, Florida, 33136, United States
The Nemours Children's Clinic - Orlando
Orlando, Florida, 32827, United States
Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
University of Iowa
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Michigan, Michigan Medicine
Ann Arbor, Michigan, 48109, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, 55455, United States
Children's Mercy Kansas City
Kansas City, Missouri, 64108, United States
St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
The Cystic Fibrosis Center of Western New York
Buffalo, New York, 14203, United States
Children's Hospital of New York
New York, New York, 10032, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
New York Medical College at Westchester Medical Center
Valhalla, New York, 10595, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oklahoma Cystic Fibrosis Center
Oklahoma City, Oklahoma, 73104, United States
Oregon Health Sciences University
Portland, Oregon, 97239, United States
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, 17033, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
University of Texas Southwestern / Children's Health
Dallas, Texas, 75207, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Primary Children's Cystic Fibrosis Center
Salt Lake City, Utah, 84113, United States
Vermont Children's Hospital
Burlington, Vermont, 05401, United States
University of Virginia
Charlottesville, Virginia, 22903, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
University of Wisconsin
Madison, Wisconsin, 53792, United States
Biospecimen
Serum, Plasma, Buffy Coat, Urine, Stool, oropharyngeal (OP) swab, Bronchoalveolar lavage (BAL)
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Bonnie Ramsey, MD
Seattle Children's Hospital
- PRINCIPAL INVESTIGATOR
Lucas Hoffman, MD PhD
University of Washington/Seattle Children's
- PRINCIPAL INVESTIGATOR
Katie Larson Ode, MD
University of Iowa
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Associate Professor University of Washington
Study Record Dates
First Submitted
August 6, 2020
First Posted
August 11, 2020
Study Start
November 18, 2020
Primary Completion (Estimated)
December 1, 2029
Study Completion (Estimated)
December 1, 2029
Last Updated
August 12, 2024
Record last verified: 2024-08
Data Sharing
- IPD Sharing
- Will not share