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.
Not Applicable
Not yet recruiting
- Conditions
- Cystic FibrosisRespiratory - Other respiratory disorders / diseases
- Registration Number
- ACTRN12624001185550
- Lead Sponsor
- Caitlin Miles (Chief Investigator/Coordinator), Monash Health
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not yet recruiting
- Sex
- All
- Target Recruitment
- 160
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.
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
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Growth (composite outcome)[Weight (kg) - infant scale or standing scale <br>Length/height (cm) - infant length scale or stadiometer <br>Weight-for-length and BMI (kg/m2) and corresponding z scores and percentiles - calculated using online tool 'PediTools' Baseline (prior to commencing ETI/IVA therapy)<br>Every 3 months for the first two years of ETI/IVA therapy <br>2 years post commencement of ETI/IVA therapy ];Nutritional biomarkers (composite outcome) [Serum levels of vitamin A, D, E, B12, holoTC, ferritin, INR and CRP. Baseline (prior to commencing ETI/IVA therapy)<br>1 months post commencement of ETI/IVA therapy <br>2 years post commencement of ETI/IVA therapy ]
- Secondary Outcome Measures
Name Time Method