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

Last updated: June 5, 2025
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Active - Not Recruiting

Phase

N/A

Condition

Lung Disease

Cystic Fibrosis

Scar Tissue

Treatment

N/A

Clinical Study ID

NCT06506773
VX24-445-130
  • Ages < 6
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

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 productlabels) and are not receiving Kalydeco or any other cystic fibrosis transmembraneconductance regulator gene (CFTR) modulator

Exclusion

Key Exclusion Criteria:

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

Other protocol defined Inclusion/Exclusion criteria apply.

Study Design

Total Participants: 79
Study Start date:
July 09, 2024
Estimated Completion Date:
August 31, 2026

Connect with a study center

  • The Kids Research Institute Australia

    Nedlands,
    Australia

    Site Not Available

  • Women's and Children's Hospital

    North Adelaide,
    Australia

    Site Not Available

  • Queensland Children's Hospital

    South Brisbane,
    Australia

    Site Not Available

  • McGill University Health Centre, Glen Site, Montreal Children's Hospital

    Montreal,
    Canada

    Site Not Available

  • Centre Hospitalier de Quebec - Universite Laval

    Quebec City,
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto,
    Canada

    Site Not Available

  • British Columbia Children's Hospital

    Vancouver,
    Canada

    Site Not Available

  • Fakultni nemocnice v Motole

    Praha 5,
    Czechia

    Site Not Available

  • Charite Paediatric Pulmonology Department

    Berlin,
    Germany

    Site Not Available

  • Kinderklinik III, Abt. fur Pneumologie

    Essen,
    Germany

    Site Not Available

  • Klinikum Westbrandenburg (CF)

    Potsdam,
    Germany

    Site Not Available

  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

    Firenze,
    Italy

    Site Not Available

  • Canterbury District Health Board

    Christchurch,
    New Zealand

    Site Not Available

  • Starship Children's Hospital

    Grafton,
    New Zealand

    Site Not Available

  • Pediatric Hospital Polanki named of Maciej Płażyński

    Gdańsk,
    Poland

    Site Not Available

  • Hospital Universitario 12 de Octubre

    Madrid,
    Spain

    Site Not Available

  • Hospital Universitario Ramon y Cajal

    Madrid,
    Spain

    Site Not Available

  • Hospital Universitario Virgen del Rocio

    Sevilla,
    Spain

    Site Not Available

  • Kinderspital Zurich

    Zurich,
    Switzerland

    Site Not Available

  • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children

    Bristol,
    United Kingdom

    Site Not Available

  • Royal Hospital for Children

    Glasgow,
    United Kingdom

    Site Not Available

  • Leeds Regional Children's Cystic Fibrosis Centre

    Leeds,
    United Kingdom

    Site Not Available

  • Leicester Royal Infirmary, University of Leicester

    Leicester,
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children

    London,
    United Kingdom

    Site Not Available

  • The Royal London Children's Hospital

    London,
    United Kingdom

    Site Not Available

  • Royal Manchester Children's Hospital

    Manchester,
    United Kingdom

    Site Not Available

  • Nottingham University Hospitals NHS Trust, Children's Clinical Research Facility

    Nottingham,
    United Kingdom

    Site Not Available

  • Royal Stoke University Hospital

    Stoke on Trent,
    United Kingdom

    Site Not Available

  • Johns Hopkins All Children's Hospital Outpatient Care Center

    Saint Petersburg, Florida 33701
    United States

    Site Not Available

  • St. Luke's Cystic Fibrosis Center of Idaho

    Boise, Idaho 83702
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Boston Childrens Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Michigan Medicine

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota 55404
    United States

    Site Not Available

  • Billings Clinic

    Billings, Montana 59101
    United States

    Site Not Available

  • Children's Nebraska

    Omaha, Nebraska 68114
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Akron Children's Hospital

    Akron, Ohio 44308
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

    Cleveland, Ohio 44106
    United States

    Site Not Available

  • Dayton Children's Hospital

    Dayton, Ohio 45404
    United States

    Site Not Available

  • ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center

    Toledo, Ohio 43606
    United States

    Site Not Available

  • Prisma Health Clinical Research Unit - Midlands

    Columbia, South Carolina 29203
    United States

    Site Not Available

  • Sanford Children's Speciality Clinic

    Sioux Falls, South Dakota 57105
    United States

    Site Not Available

  • The University of Texas Health Science Center at Tyler

    Tyler, Texas 75708
    United States

    Site Not Available

  • Virginia Commonwealth University Hospital Systems, Children's Pavilion

    Richmond, Virginia 23298
    United States

    Site Not Available

  • Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

    Spokane, Washington 99204
    United States

    Site Not Available

  • West Virginia University

    Morgantown, West Virginia 26506
    United States

    Site Not Available

  • Medical College of Wisconsin

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.