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Trial registered on ANZCTR


Registration number
ACTRN12624001185550
Ethics application status
Approved
Date submitted
12/09/2024
Date registered
27/09/2024
Date last updated
27/09/2024
Date data sharing statement initially provided
27/09/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Pancreatic, nutritional and clinical outcomes in children 0-5 years with cystic fibrosis during the first 2 years of CFTR modulator therapy (PaNC): a multicentre study.
Scientific title
Pancreatic, nutritional and clinical outcomes in children 0-5 years with cystic fibrosis during the first 2 years of CFTR modulator therapy (PaNC): a multicentre prospective observational study.
Secondary ID [1] 311748 0
None
Universal Trial Number (UTN)
Trial acronym
PaNC
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 333236 0
Condition category
Condition code
Respiratory 329923 329923 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Nutritional, pancreatic and clinical outcomes in children 0-5 years with cystic fibrosis during the first 2 years of elexacaftor/tezacaftor/ivacaftor (ETI) or ivacaftor (IVA) treatment.

Participant involvement: Written informed consent from the child’s legal parent/guardian will be obtained prior to enrolment in the PaNC study. The child's demographic and health-related data will be accessed by study investigators from data which is collected as part of routine CF care. Data sources will include electronic or paper medical records sources. The child and legal parent/guardian will be reviewed by CF clinic dietitians, as is part of routine CF care. The child's routine investigations, such as annual nutritional bloods and stool samples for faecal elastase, will be accessed for inclusion in the PaNC study.
Intervention code [1] 328203 0
Not applicable
Comparator / control treatment
Comparisons will be made between data collected in this study and established literature on children 2-5 years of age who are not treated with ETI or IVA and are receiving established routine CF treatment, however control data for children 2-5 years of age who are not treated with ETI or IVA and are receiving established routine CF treatment will not be collected as part of study procedures.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 337689 0
Growth (composite outcome)
Timepoint [1] 337689 0
Baseline (prior to commencing ETI/IVA therapy)
Every 3 months for the first two years of ETI/IVA therapy
2 years post commencement of ETI/IVA therapy
Primary outcome [2] 339353 0
Nutritional biomarkers (composite outcome)
Timepoint [2] 339353 0
Baseline (prior to commencing ETI/IVA therapy)
1 months post commencement of ETI/IVA therapy
2 years post commencement of ETI/IVA therapy
Secondary outcome [1] 432851 0
Exocrine pancreatic function
Timepoint [1] 432851 0
Baseline (prior to commencing ETI/IVA therapy)
Every 6 months for the first two years of ETI/IVA therapy
2 years post commencement of ETI/IVA therapy
Secondary outcome [2] 432852 0
Medication burden (composite outcome)
Timepoint [2] 432852 0
Baseline (prior to commencing ETI/IVA therapy)
Every 3 months for the first two years of ETI/IVA therapy
2 years post commencement of ETI/IVA therapy
Secondary outcome [3] 432855 0
Oral or enteral nutrition support (composite outcome)
Timepoint [3] 432855 0
Baseline (prior to commencing ETI/IVA therapy)
Every 3 months for the first two years of ETI/IVA therapy
2 years post commencement of ETI/IVA therapy
Secondary outcome [4] 439627 0
Sweat chloride
Timepoint [4] 439627 0
Baseline (prior to commencing ETI/IVA therapy)
1 months post commencement of ETI/IVA therapy
2 years post commencement of ETI/IVA therapy

Eligibility
Key inclusion criteria
Infants and children 4 months - 5 years of age with a confirmed diagnosis of CF (as diagnosed by standard criteria; sweat test greater than or equal to 60mEq/L and/or CFTR gene mutation analysis) who are eligible for the commencement of ETI or IVA. Infants and children will be eligible for the study regardless of their respiratory health or presence of comorbidities.
Minimum age
4 Months
Maximum age
5 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Infants and children older than 5 years of age with a confirmed diagnosis of CF or infants and children 4 months - 5 years of age who are not eligible for the commencement of ETI or IVA.

Study design
Purpose
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
30/09/2024
Actual
Date of last participant enrolment
Anticipated
30/03/2025
Actual
Date of last data collection
Anticipated
30/03/2027
Actual
Sample size
Target
160
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA,VIC

Funding & Sponsors
Funding source category [1] 316080 0
Hospital
Name [1] 316080 0
Monash Health
Country [1] 316080 0
Australia
Primary sponsor type
Individual
Name
Caitlin Miles (Chief Investigator/Coordinator), Monash Health
Address
Country
Australia
Secondary sponsor category [1] 319749 0
None
Name [1] 319749 0
Address [1] 319749 0
Country [1] 319749 0
Other collaborator category [1] 283205 0
Hospital
Name [1] 283205 0
Monash Health
Address [1] 283205 0
Country [1] 283205 0
Australia
Other collaborator category [2] 283206 0
University
Name [2] 283206 0
Monash University
Address [2] 283206 0
Country [2] 283206 0
Australia
Other collaborator category [3] 283207 0
Hospital
Name [3] 283207 0
Royal Children's Hospital
Address [3] 283207 0
Country [3] 283207 0
Australia
Other collaborator category [4] 283208 0
Hospital
Name [4] 283208 0
Queensland Children's Hospital and Health Service
Address [4] 283208 0
Country [4] 283208 0
Australia
Other collaborator category [5] 283209 0
Hospital
Name [5] 283209 0
Sydney Children's Hospital Network
Address [5] 283209 0
Country [5] 283209 0
Australia
Other collaborator category [6] 283210 0
Hospital
Name [6] 283210 0
John Hunter Children's Hospital
Address [6] 283210 0
Country [6] 283210 0
Australia
Other collaborator category [7] 283211 0
Hospital
Name [7] 283211 0
The Women's and Children's Hospital
Address [7] 283211 0
Country [7] 283211 0
Australia
Other collaborator category [8] 283212 0
Hospital
Name [8] 283212 0
Perth Children's Hospital
Address [8] 283212 0
Country [8] 283212 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 314899 0
Monash Health Human Research Ethics Committee A
Ethics committee address [1] 314899 0
https://monashhealth.org/research/resources/resource-library/
Ethics committee country [1] 314899 0
Australia
Date submitted for ethics approval [1] 314899 0
07/03/2024
Approval date [1] 314899 0
04/04/2024
Ethics approval number [1] 314899 0
RES-24-0000-113A

Summary
Brief summary
Hypothesis: Infants and young children 4 months to 5 years of age who are commenced on ETI or IVA will have improved and sustained growth and nutritional outcomes, exocrine pancreatic function and sweat chloride levels. Primary aim: To evaluate longitudinal patterns in growth and nutrition in children 4 months to 5 years of age with CF during the first 2 years of treatment with ETI or IVA. Secondary aim: To describe changes in pancreatic function, clinical outcomes and nutritional interventions in children 4 months to 5 years of age with CF during the first 2 years of treatment with ETI or IVA. Longitudinal, ‘real world’ studies are required to better understand the breadth and significance of CF exocrine pancreatic involvement in children with CF who are exposed to early CFTR correction as infants and children and to better understand the nutritional implications.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 133094 0
Ms Caitlin Miles
Address 133094 0
Allied Health, Monash Children's Hospital, Level 2, 246 Clayton Road, Clayton VIC 3168
Country 133094 0
Australia
Phone 133094 0
+613 8572 3003
Fax 133094 0
Email 133094 0
[email protected]
Contact person for public queries
Name 133095 0
Caitlin Miles
Address 133095 0
Allied Health, Monash Children's Hospital, Level 2, 246 Clayton Road, Clayton VIC 3168
Country 133095 0
Australia
Phone 133095 0
+613 8572 3003
Fax 133095 0
Email 133095 0
[email protected]
Contact person for scientific queries
Name 133096 0
Caitlin Miles
Address 133096 0
Allied Health, Monash Children's Hospital, Level 2, 246 Clayton Road, Clayton VIC 3168
Country 133096 0
Australia
Phone 133096 0
+613 8572 3003
Fax 133096 0
Email 133096 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.