Cystic FibrosisNCT06506773
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
An observational study of Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 50 sites in 11 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 6 months
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 79 participants
- Where
- 50 sitesin 11 countries
- Study dates
- 9 Jul 2024to 27 Nov 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 6 months
- All sexes
- No healthy volunteers
- Child
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".
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
50 sites in 11 countries.
50 sites in 11 countries
United States22 sites
Akron Children's Hospital
Akron, Ohio, 44308
Michigan Medicine
Ann Arbor, Michigan, 48109
Billings Clinic
Billings, Montana, 59101
St. Luke's Cystic Fibrosis Center of Idaho
Boise, Idaho, 83702
Boston Childrens Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Prisma Health Clinical Research Unit - Midlands
Columbia, South Carolina, 29203
Dayton Children's Hospital
Dayton, Ohio, 45404
Duke University Medical Center
Durham, North Carolina, 27710
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
West Virginia University
Morgantown, West Virginia, 26506
Children's Nebraska
Omaha, Nebraska, 68114
Virginia Commonwealth University Hospital Systems, Children's Pavilion
Richmond, Virginia, 23298
Sanford Children's Speciality Clinic
Sioux Falls, South Dakota, 57105
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
Spokane, Washington, 99204
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, 33701
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
Toledo, Ohio, 43606
The University of Texas Health Science Center at Tyler
Tyler, Texas, 75708
United Kingdom9 sites
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children
Bristol
Royal Hospital for Children
Glasgow
Leeds Regional Children's Cystic Fibrosis Centre
Leeds
Leicester Royal Infirmary, University of Leicester
Leicester
Great Ormond Street Hospital for Children
London
The Royal London Children's Hospital
London
Royal Manchester Children's Hospital
Manchester
Nottingham University Hospitals NHS Trust, Children's Clinical Research Facility
Nottingham
Royal Stoke University Hospital
Stoke-on-Trent
Canada4 sites
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal
Centre Hospitalier de Quebec - Universite Laval
Québec
The Hospital for Sick Children
Toronto
British Columbia Children's Hospital
Vancouver
Australia3 sites
The Kids Research Institute Australia
Nedlands
Women's and Children's Hospital
North Adelaide
Queensland Children's Hospital
South Brisbane
Germany3 sites
Charite Paediatric Pulmonology Department
Berlin
Kinderklinik III, Abt. fur Pneumologie
Essen
Klinikum Westbrandenburg (CF)
Potsdam
Spain3 sites
Hospital Universitario 12 de Octubre
Madrid
Hospital Universitario Ramon y Cajal
Madrid
Hospital Universitario Virgen del Rocio
Seville
New Zealand2 sites
Canterbury District Health Board
Christchurch
Starship Children's Hospital
Grafton
Italy1 site
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
Florence
About this study
Official title: 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.
- Conditions
- Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Participants with FE-1 greater than or equal to (≥) 200 microgram per gram (µg/g) Over Time
Measured: From Enrollment up to the Infant turning 12 Months of Age
Secondary outcomes (1)
FE-1 level Over Time
Measured: From Enrollment up to the Infant turning 12 Months of Age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2024
- Study start9 Jul 2024
- Primary completion27 Nov 2025
- Study completion27 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 79 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06506773
- Sponsor's protocol ID
- VX24-445-130
- Status last verified
- Dec 2025
- First submitted
- 27 Jun 2024
- Last updated
- 31 Dec 2025
- Results posted
- No
- Individual data sharing
- No: 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/
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis Less Than 12 Months of Age
- ClinicalTrials.gov ID
- NCT06506773
- Acronym / sponsor ID
- VX24-445-130
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 79 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 9 Jul 2024 (actual)
- Primary completion
- 27 Nov 2025 (actual)
- Study completion
- 27 Nov 2025 (actual)
- First posted
- 17 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort A | – | –Observational follow-up study to characterize the natural history of exocrine pancreatic function by measuring FE-1 in infants with CF. |
Who may qualify
- Ages
- Up to 6 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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".
Key inclusion criteria (2 listed)
- 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 (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
1 secondary outcome
FE-1 level Over Time
Time frame: From Enrollment up to the Infant turning 12 Months of Age
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2025.
- Individual participant dataSharing planned: No. 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/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
50 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 50 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, United Kingdom, Canada, Australia, Germany and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Dec 2025.