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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06506773




Registration number
NCT06506773
Ethics application status
Date submitted
27/06/2024
Date registered
17/07/2024

Titles & IDs
Public title
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
Scientific title
A Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis Less Than 12 Months of Age
Secondary ID [1] 0 0
VX24-445-130
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Cohort A - Observational follow-up study to characterize the natural history of exocrine pancreatic function by measuring FE-1 in infants with CF.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of Participants with FE-1 greater than or equal to (=) 200 microgram per gram (µg/g) Over Time
Timepoint [1] 0 0
From Enrollment up to the Infant turning 12 Months of Age
Secondary outcome [1] 0 0
FE-1 level Over Time
Timepoint [1] 0 0
From Enrollment up to the Infant turning 12 Months of Age

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
No limit
Maximum age
6 Months
Sex
Both males and females
Can healthy volunteers participate?
No
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 design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/07/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2026
Actual
Sample size
Target
150
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Kids Research Institute Australia - Nedlands
Recruitment hospital [2] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment hospital [3] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
- Nedlands
Recruitment postcode(s) [2] 0 0
- North Adelaide
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Idaho
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Montana
Country [9] 0 0
United States of America
State/province [9] 0 0
Nebraska
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
South Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Virginia
Country [15] 0 0
United States of America
State/province [15] 0 0
Washington
Country [16] 0 0
United States of America
State/province [16] 0 0
West Virginia
Country [17] 0 0
United States of America
State/province [17] 0 0
Wisconsin
Country [18] 0 0
Canada
State/province [18] 0 0
Montreal
Country [19] 0 0
Canada
State/province [19] 0 0
Quebec City
Country [20] 0 0
Canada
State/province [20] 0 0
Toronto
Country [21] 0 0
Canada
State/province [21] 0 0
Vancouver
Country [22] 0 0
Czechia
State/province [22] 0 0
Praha 5
Country [23] 0 0
Germany
State/province [23] 0 0
Berlin
Country [24] 0 0
Germany
State/province [24] 0 0
Essen
Country [25] 0 0
Germany
State/province [25] 0 0
Potsdam
Country [26] 0 0
New Zealand
State/province [26] 0 0
Christchurch
Country [27] 0 0
New Zealand
State/province [27] 0 0
Grafton
Country [28] 0 0
Poland
State/province [28] 0 0
Gdansk
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Spain
State/province [30] 0 0
Sevilla
Country [31] 0 0
Switzerland
State/province [31] 0 0
Zurich
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Bristol
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Glasgow
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Leeds
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Leicester
Country [36] 0 0
United Kingdom
State/province [36] 0 0
London
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Manchester
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Nottingham
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Stoke on Trent

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Information
Address 0 0
Country 0 0
Phone 0 0
617-341-6777
Fax 0 0
Email 0 0
medicalinfo@vrtx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06506773