Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Function After Highly Effective Modulator Therapy

COMPLETED

NCT07632768

PHASE4

Sponsor: Indiana University

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

Official title: The Return of the Pancreas: Evaluating Impact of CFTR Modulators on Pancreatic Function

Key Details

Gender

All

Age Range

0 Years - 18 Years

Study Type

INTERVENTIONAL

Enrollment

17

Start Date

2024-12-03

Completion Date

2026-01-21

Last Updated

2026-07-02

Healthy Volunteers

No

Conditions

Cystic Fibrosis (CF)Pancreatic Insufficiency Pancreas Disease Exocrine Pancreatic Insufficiency Exocrine Pancreatic Insufficiency in Subjects With Cystic Fibrosis CFTR Gene Mutation

Interventions

DRUG

Discontinuation of Pancreatic Enzyme Replacement Therapy

Participants with cystic fibrosis who demonstrate pancreatic sufficiency, defined as fecal elastase-1 (FE-1) ≥200 µg/g after treatment with CFTR modulator therapy, will discontinue pancreatic enzyme replacement therapy (PERT) under medical supervision. Participants will undergo follow-up assessments over 6 months, including monitoring of growth, gastrointestinal symptoms, nutritional laboratory markers, and repeat fecal elastase testing to evaluate the safety and sustainability of pancreatic function recovery after PERT discontinuation.

Locations (1)

Riley Hospital for Children

Indianapolis, Indiana, United States

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