Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children (BEGIN)
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|ClinicalTrials.gov Identifier: NCT04509050|
Recruitment Status : Recruiting
First Posted : August 11, 2020
Last Update Posted : May 6, 2023
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|Condition or disease||Intervention/treatment|
|Cystic Fibrosis||Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor|
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact in children with cystic fibrosis (CF) on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health.
Total duration of the study is expected to be 6 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 6 visits over a period of up to 3 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 one "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and five "after ivacaftor or elex/tez/iva" visits over a 24-month follow-up period.
|Study Type :||Observational|
|Estimated Enrollment :||210 participants|
|Official Title:||A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)|
|Actual Study Start Date :||November 18, 2020|
|Estimated Primary Completion Date :||October 1, 2025|
|Estimated Study Completion Date :||October 1, 2025|
Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
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.
Drug: 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
- Part A Primary Outcome Measure: Change in weight-for-age z-scores [ Time Frame: Baseline to 12 months ]Weight-for-age z-scores over time
- Part A Primary Outcome Measure: Change in height-for-age z-scores [ Time Frame: Baseline to 12 months ]Height-for-age z-scores over time
- Part B Primary Outcome Measure: Change in weight-for-age z-scores [ Time Frame: 1, 3, 6, 12, and 24 months ]Change in weight-for-age z-scores from baseline
- Part B Primary Outcome Measure: Change in height-for-age z-scores [ Time Frame: 1, 3, 6, 12, and 24 months ]Change in height-for-age z-scores from baseline
Biospecimen Retention: Samples With DNA
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|Ages Eligible for Study:||up to 5 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
Part A - children with confirmed diagnosis of cystic fibrosis who are less than 5 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 6 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy
- Less than 5 years of age at the first study visit.
- Documentation of a CF diagnosis.
- 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.
- 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 180 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04509050
|Contact: Rachael Buckinghamfirstname.lastname@example.org|
|Contact: Nicole Rogersemail@example.com|
|Principal Investigator:||Bonnie Ramsey, MD||Seattle Children's|
|Principal Investigator:||Lucas Hoffman, MD PhD||University of Washington/Seattle Children's|
|Principal Investigator:||Michael Stalvey, MD||University of Alabama at Birmingham|
|Responsible Party:||Sonya Heltshe, Associate Professor University of Washington, Seattle Children's Hospital|
|Other Study ID Numbers:||
|First Posted:||August 11, 2020 Key Record Dates|
|Last Update Posted:||May 6, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
triple combination therapy
Digestive System Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action