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

NCT07632768 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2026-07-02

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • Indiana Clinical and Translational Sciences Institute

    collaborator OTHER

  • Indiana University

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-03
Primary Completion
2026-01-21
Completion
2026-01-21
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07632768 on ClinicalTrials.gov