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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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 Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 9, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 27, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 27, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 27, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 16, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 17, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 31, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 26, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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