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

Sonya Heltshe

Status: Enrolling

Conditions: Cystic Fibrosis

Treatments: Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor

Study type: Observational

Funder types: Other

Identifiers

NCT04509050

BEGIN-OB-19

Details and patient eligibility

About

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).

Full description

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.

Enrollment: 210 estimated patients

Sex: All

Ages: Under 10 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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.

Exclusion criteria

• 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.

Trial design

210 participants in 2 patient groups

Part A

Description:

Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.

Part B

Description:

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.

Treatment:

Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor