Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

Официальное название

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

Обзор

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).

Подробное описание

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 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.

Вмешательства

• Препарат 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

Первичные конечные точки

• Part A Primary Outcome Measure: Change in weight-for-age z-scores [Срок оценки: Baseline to 12 months]
• Part A Primary Outcome Measure: Change in height-for-age z-scores [Срок оценки: Baseline to 12 months]
• Part B Primary Outcome Measure: Change in weight-for-age z-scores [Срок оценки: Baseline to 12 months]
• Part B Primary Outcome Measure: Change in height-for-age z-scores [Срок оценки: Baseline to 12 months]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 35 центров

• The Children's Hospital Alabama, University of Alabama at Birmingham — Birmingham
• Stanford University Medical Center — Palo Alto
• Children's Hospital Colorado — Aurora
• Nemours Children's Clinic — Jacksonville
• University of Miami — Miami
• The Nemours Children's Clinic - Orlando — Orlando
• Riley Hospital for Children — Indianapolis
• University of Iowa — Iowa City
• … и ещё 27 центров

Идентификаторы

NCT: NCT04509050 · BEGIN-OB-19

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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