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

NCT ID: NCT06506773

Last Updated: 2025-12-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

79 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-07-09

Study Completion Date

2025-11-27

Brief Summary

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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.

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Cohort A

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

No interventions assigned to this group

Eligibility Criteria

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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

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
Maximum Eligible Age

6 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Johns Hopkins All Children's Hospital Outpatient Care Center

St. Petersburg, Florida, United States

Site Status

St. Luke's Cystic Fibrosis Center of Idaho

Boise, Idaho, United States

Site Status

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

Chicago, Illinois, United States

Site Status

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

Site Status

Boston Childrens Hospital

Boston, Massachusetts, United States

Site Status

Michigan Medicine

Ann Arbor, Michigan, United States

Site Status

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

Minneapolis, Minnesota, United States

Site Status

Billings Clinic

Billings, Montana, United States

Site Status

Children's Nebraska

Omaha, Nebraska, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Akron Children's Hospital

Akron, Ohio, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

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

Cleveland, Ohio, United States

Site Status

Dayton Children's Hospital

Dayton, Ohio, United States

Site Status

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

Toledo, Ohio, United States

Site Status

Prisma Health Clinical Research Unit - Midlands

Columbia, South Carolina, United States

Site Status

Sanford Children's Speciality Clinic

Sioux Falls, South Dakota, United States

Site Status

The University of Texas Health Science Center at Tyler

Tyler, Texas, United States

Site Status

Virginia Commonwealth University Hospital Systems, Children's Pavilion

Richmond, Virginia, United States

Site Status

Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

Spokane, Washington, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

The Kids Research Institute Australia

Nedlands, , Australia

Site Status

Women's and Children's Hospital

North Adelaide, , Australia

Site Status

Queensland Children's Hospital

South Brisbane, , Australia

Site Status

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

Montreal, , Canada

Site Status

Centre Hospitalier de Quebec - Universite Laval

Québec, , Canada

Site Status

The Hospital for Sick Children

Toronto, , Canada

Site Status

British Columbia Children's Hospital

Vancouver, , Canada

Site Status

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Kinderklinik III, Abt. fur Pneumologie

Essen, , Germany

Site Status

Klinikum Westbrandenburg (CF)

Potsdam, , Germany

Site Status

Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

Florence, , Italy

Site Status

Canterbury District Health Board

Christchurch, , New Zealand

Site Status

Starship Children's Hospital

Grafton, , New Zealand

Site Status

Pediatric Hospital Polanki named of Maciej Płażyński

Gdansk, , Poland

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Kinderspital Zurich

Zurich, , Switzerland

Site Status

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

Bristol, , United Kingdom

Site Status

Royal Hospital for Children

Glasgow, , United Kingdom

Site Status

Leeds Regional Children's Cystic Fibrosis Centre

Leeds, , United Kingdom

Site Status

Leicester Royal Infirmary, University of Leicester

Leicester, , United Kingdom

Site Status

Great Ormond Street Hospital for Children

London, , United Kingdom

Site Status

The Royal London Children's Hospital

London, , United Kingdom

Site Status

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Site Status

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

Nottingham, , United Kingdom

Site Status

Royal Stoke University Hospital

Stoke-on-Trent, , United Kingdom

Site Status

Countries

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United States Australia Canada Czechia Germany Italy New Zealand Poland Spain Switzerland United Kingdom

Other Identifiers

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VX24-445-130

Identifier Type: -

Identifier Source: org_study_id