NCT07632768CompletedPhase 4
Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Function After Highly Effective Modulator Therapy
The Return of the Pancreas: Evaluating Impact of CFTR Modulators on Pancreatic Function
Source: ClinicalTrials.gov · Last updated 2 Jul 2026
Brief summary (registry, in English)
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
- Status (registry)
- Completed
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 17 (actual)
- First submitted
- 26 May 2026
- Start date
- 3 Dec 2024
- Primary completion date
- 21 Jan 2026
- Lead sponsor
- Indiana University
- Diseases
- Cystic fibrosis
- NCT number
- NCT07632768
What you commit to
- Study type: Interventional · Phase 4
- Participants enrolled: 17
- From 3 Dec 2024 to the primary completion date, 21 Jan 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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%
Age
- 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
Who the study is looking for
- 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.
Age in the registry: 0 years to 18 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Riley Hospital for Children Coordinator of this site? Confirm its status | Indianapolis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.