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

Part of paid clinical trials in Indianapolis, Indiana.

Sponsor
Indiana University
Study ID
NCT07632768
Phase
PHASE4
Status
Completed

Conditions

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

Eligibility Criteria

Sex
ALL
Age
0 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • Discontinuation of Pancreatic Enzyme Replacement Therapy — DRUG
    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.

Study Details

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

Key Dates

Start date
Dec 3, 2024
Status verified
Jun 2026
Primary completion
Jan 21, 2026
Completion
Jan 21, 2026

Study Design

Enrollment
17 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Intervention: 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.

Primary Outcome Measure

Body Mass Index (BMI) [ Time Frame: 6 months ]

Locations (1)

FacilityCityStateZIPSite coordinators
Riley Hospital for ChildrenIndianapolisIndiana46202-

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By research site
Riley Hospital for Children· Indianapolis, IN

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