Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)

Vertex Pharmaceuticals

Status: Completed

Conditions: Cystic Fibrosis

Study type: Observational

Funder types: Industry

Identifiers

NCT06506773

VX24-445-130

Details and patient eligibility

About

The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.

Enrollment: 79 patients

Sex: All

Ages: Under 6 months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants with CF less than (<) 6 months of age at the index date
• Participants not eligible to receive commercial Kalydeco™ (based on local product labels) and are not receiving Kalydeco or any other cystic fibrosis transmembrane conductance regulator gene (CFTR) modulator

Key Exclusion Criteria:

• Participant whose mother took any CFTR modulator while pregnant with the participant, or who has any history of exposure to a CFTR modulator

Other protocol defined Inclusion/Exclusion criteria apply.

Trial design

79 participants in 1 patient group

Cohort A

Description:

Observational follow-up study to characterize the natural history of exocrine pancreatic function by measuring FE-1 in infants with CF.