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

December 26, 2025 updated by: Vertex Pharmaceuticals Incorporated

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

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

79

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Nedlands, Australia
        • The Kids Research Institute Australia
      • North Adelaide, Australia
        • Women's and Children's Hospital
      • South Brisbane, Australia
        • Queensland Children's Hospital
  • Canada
      • Montreal, Canada
        • McGill University Health Centre, Glen Site, Montreal Children's Hospital
      • Québec, Canada
        • Centre Hospitalier de Quebec - Universite Laval
      • Toronto, Canada
        • The Hospital for Sick Children
      • Vancouver, Canada
        • British Columbia Children's Hospital
  • Czechia
      • Prague, Czechia
        • Fakultni nemocnice v Motole
  • Germany
      • Berlin, Germany
        • Charite Paediatric Pulmonology Department
      • Essen, Germany
        • Kinderklinik III, Abt. fur Pneumologie
      • Potsdam, Germany
        • Klinikum Westbrandenburg (CF)
  • Italy
      • Florence, Italy
        • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
  • New Zealand
      • Christchurch, New Zealand
        • Canterbury District Health Board
      • Grafton, New Zealand
        • Starship Children's Hospital
  • Poland
      • Gdansk, Poland
        • Pediatric Hospital Polanki named of Maciej Płażyński
  • Spain
      • Madrid, Spain
        • Hospital Universitario 12 de Octubre
      • Madrid, Spain
        • Hospital Universitario Ramon y Cajal
      • Seville, Spain
        • Hospital Universitario Virgen del Rocio
  • Switzerland
      • Zurich, Switzerland
        • Kinderspital Zürich
  • United Kingdom
      • Bristol, United Kingdom
        • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children
      • Glasgow, United Kingdom
        • Royal Hospital For Children
      • Leeds, United Kingdom
        • Leeds Regional Children's Cystic Fibrosis Centre
      • Leicester, United Kingdom
        • Leicester Royal Infirmary, University of Leicester
      • London, United Kingdom
        • Great Ormond Street Hospital for Children
      • London, United Kingdom
        • The Royal London Children's Hospital
      • Manchester, United Kingdom
        • Royal Manchester Children's Hospital
      • Nottingham, United Kingdom
        • Nottingham University Hospitals NHS Trust, Children's Clinical Research Facility
      • Stoke-on-Trent, United Kingdom
        • Royal Stoke University Hospital
  • United States
    • Florida
      • St. Petersburg, Florida, United States, 33701
        • Johns Hopkins All Children's Hospital Outpatient Care Center
    • Idaho
      • Boise, Idaho, United States, 83702
        • St. Luke's Cystic Fibrosis Center of Idaho
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children at Indiana University Health
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Childrens Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Michigan Medicine
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
    • Montana
      • Billings, Montana, United States, 59101
        • Billings Clinic
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Children's Nebraska
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Ohio
      • Akron, Ohio, United States, 44308
        • Akron Children's Hospital
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cleveland, Ohio, United States, 44106
        • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
      • Dayton, Ohio, United States, 45404
        • Dayton Children's Hospital
      • Toledo, Ohio, United States, 43606
        • ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Prisma Health Clinical Research Unit - Midlands
    • South Dakota
      • Sioux Falls, South Dakota, United States, 57105
        • Sanford Children's Speciality Clinic
    • Texas
      • Tyler, Texas, United States, 75708
        • The University of Texas Health Science Center at Tyler
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University Hospital Systems, Children's Pavilion
    • Washington
      • Spokane, Washington, United States, 99204
        • Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
        • West Virginia University
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Infants with CF less than (<) 6 months of age at the index date who are not eligible for commercial Kalydeco™ (according to local product labels). The day of enrollment into this study will be referred to as the "index date".

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
From Enrollment up to the Infant turning 12 Months of Age

Secondary Outcome Measures

Outcome Measure
Time Frame
FE-1 level Over Time
Time Frame: From Enrollment up to the Infant turning 12 Months of Age
From Enrollment up to the Infant turning 12 Months of Age

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 9, 2024

Primary Completion (Actual)

November 27, 2025

Study Completion (Actual)

November 27, 2025

Study Registration Dates

First Submitted

June 27, 2024

First Submitted That Met QC Criteria

July 16, 2024

First Posted (Actual)

July 17, 2024

Study Record Updates

Last Update Posted (Actual)

December 31, 2025

Last Update Submitted That Met QC Criteria

December 26, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX24-445-130

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https: https://www.vrtx.com/our-science/clinical-trials-data-sharing/

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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