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

NCT ID NCT07632768

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for CFTR GENE MUTATION are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.