Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
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
Date last updated
21/11/2024

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
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Montana
Country [8] 0 0
United States of America
State/province [8] 0 0
Nebraska
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
South Carolina
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Virginia
Country [14] 0 0
United States of America
State/province [14] 0 0
Washington
Country [15] 0 0
United States of America
State/province [15] 0 0
West Virginia
Country [16] 0 0
Canada
State/province [16] 0 0
Quebec City
Country [17] 0 0
Canada
State/province [17] 0 0
Toronto
Country [18] 0 0
Canada
State/province [18] 0 0
Vancouver
Country [19] 0 0
Czechia
State/province [19] 0 0
Praha 5
Country [20] 0 0
Germany
State/province [20] 0 0
Berlin
Country [21] 0 0
Germany
State/province [21] 0 0
Essen
Country [22] 0 0
New Zealand
State/province [22] 0 0
Grafton
Country [23] 0 0
Poland
State/province [23] 0 0
Gdansk
Country [24] 0 0
Spain
State/province [24] 0 0
Madrid
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Bristol
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Glasgow
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Leeds
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Leicester
Country [29] 0 0
United Kingdom
State/province [29] 0 0
London
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Manchester
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Nottingham
Country [32] 0 0
United Kingdom
State/province [32] 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
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.
Trial website
https://clinicaltrials.gov/study/NCT06506773
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
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06506773