Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Fun… (NCT07632768) | Clinical Trial Compass
CompletedPhase 4
Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Function After Highly Effective Modulator Therapy
United States17 participantsStarted 2024-12-03
Plain-language summary
The goal of this clinical trial is to evaluate the safety of stopping pancreatic enzyme replacement therapy (PERT) in children with cystic fibrosis (CF) receiving CFTR modulator therapy (CFTRm) who have regained pancreatic sufficiency. The main questions it aims to answer are:
1. Does discontinuation of PERT affect gastrointestinal symptoms, nutritional status, pancreatic function, or body composition over 6 months?
2. Does stopping PERT increase gastrointestinal symptoms or affect nutritional status, pancreatic function, or body composition compared with continuing PERT?
Researchers will evaluate changes in growth, gastrointestinal symptoms, vitamin levels, pancreatic function, and body composition following PERT discontinuation.
Participants will:
* Complete study visits and assessments over 6 months
* Continue or discontinue PERT based on study assignment
* Undergo anthropometric measurements
* Complete questionnaires about gastrointestinal symptoms
* Provide blood samples to assess vitamin levels and coagulation markers
* Provide stool samples to measure fecal elastase-1 (FE-1) and evaluate pancreatic function
Who can participate
Age range
0 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial looked at stopping pancreatic enzyme replacement therapy in kids with CF whose pancreatic function improved on highly effective modulator therapy — is my child's pancreatic function recovering enough that this could even be a conversation worth having with our team?
2Since the trial measured BMI, weight, and height as its main outcomes, what would a concerning drop in any of those numbers look like in my child, and how quickly would we catch it if stopping enzymes turned out to be the wrong call?
3The trial is already completed — has the data been published yet, and can you walk me through what the results actually showed about whether it was safe for these kids to stop enzyme replacement?
4Given that this was a Phase 4 trial — meaning it was studying a treatment already in use — how does that affect how confident you are in the findings compared to earlier-phase research?
5Even if stopping enzymes seems safe based on this trial's data, are there reasons specific to my child's situation — like their particular CFTR mutation or nutritional history — that would make you want to keep them on enzyme therapy regardless?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.