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.

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.

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ANZCTR

Registry ID

ACTRN12624001185550

Acronym

PaNC

Enrollment

Registered

2024-09-27

Start date

2024-10-18

Completion date

2025-04-01

Last updated

2025-09-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

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.

Interventions

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.

Eligibility

Sex/Gender

All

Age

4 Months to 5 Years

Healthy volunteers

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.

Outcome results