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This study assessed whether it is safe to stop pancreatic enzyme replacement therapy in children with cystic fibrosis whose pancreatic function improved after CFTR modulator therapy. It involved discontinuing pancreatic enzyme replacement therapy and could include children aged 18 or younger who previously had pancreatic insufficiency but regained pancreatic function while using a CFTR modulator (such as ivacaftor or elexacaftor/tezacaftor/ivacaftor). It was carried out in Phase 4 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Diagnosis of cystic fibrosis. * History of pancreatic insufficiency, documented by a prior fecal elastase-1 (FE-1) concentration \<200 µg/g stool. * Current pancreatic sufficiency at study entry, defined as fecal elastase-1 (FE-1) concentration ≥200 µg/g stool after treatment with a CFTR modulator. * Age ≤18 years. * Current use of a CFTR modulator, including ivacaftor, elexacaftor/tezacaftor/ivacaftor, or vanzacaftor/tezacaftor/deutivacaftor. Exclusion Criteria: * CF-related diabetes requiring current insulin use * Advanced CF liver disease as defined by nodular liver, advanced fibrosis (F4), multi-lobular cirrhosis with or without portal hypertension, non-cirrhotic portal hypertension * Short gut syndrome as defined by need for surgical bowel resection and subsequent need for parenteral nutrition for \> 60 days or bowel length less than 25% * Moderate to severe malnutrition, defined as a BMI-for-age z score ≤ -2 for participants aged ≥2 years or a weight-for-length z score ≤ -2 for participants aged \<2 years