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

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

Up to 6 month

Sex eligibility

All sexes

Study type

Observational

Primary location

The Kids Research Institute Australia, Nedlands, Australia

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

Proportion of Participants with FE-1 greater than or equal to (≥) 200 microgram per gram (µg/g) Over Time

Time frame: From Enrollment up to the Infant turning 12 Months of Age

Secondary outcomes

FE-1 level Over Time

Time frame: From Enrollment up to the Infant turning 12 Months of Age

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis Less Than 12 Months of Age

Important dates

Study start: 2024
Primary completion: 2025
Study completion: 2025
First posted: Jul 17, 2024
Registry last updated: Dec 31, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed