CF breakthrough: kids may ditch daily enzyme pills after modulator treatment

NCT ID NCT07632768

Disease control Sponsor: Indiana University Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This study looked at whether children with cystic fibrosis (CF) who are taking CFTR modulator drugs and have regained normal pancreatic function can safely stop taking pancreatic enzyme replacement therapy (PERT). Seventeen children were followed for six months after stopping enzymes. Researchers monitored their growth, nutrition, and digestive symptoms to see if stopping enzymes caused any problems.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: CFTR modulator therapy (ivacaftor, elexacaftor/tezacaftor/ivacaftor, or vanzacaftor/tezacaftor/deutivacaftor)
What this could lead to: If successful, this could show that some children with CF can safely stop taking daily pancreatic enzyme pills, simplifying their treatment and improving quality of life.
What could go wrong: This is a small, completed study with only 17 participants, so results may not apply to everyone. Stopping enzymes could cause nutritional problems or worsen symptoms in some children.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

17 people

The number who actually took part.

Started

Dec 2024

Finished

Jan 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

cystic fibrosis exocrine pancreatic insufficiency pancreas disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CFTR gene mutation Cystic fibrosis (CF) Exocrine pancreatic insufficiency Exocrine pancreatic insufficiency in subjects with cystic fibrosis Pancreas disease Pancreatic insufficiency

CF breakthrough: kids may ditch daily enzyme pills after modulator treatment

What the study statuses mean