Gene therapy trial for cystic fibrosis halted early

NCT ID NCT06515002

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a single dose of an inhaled gene therapy called BI 3720931 in adults with cystic fibrosis who cannot take standard CFTR modulator drugs. The trial was in two phases and included only 5 participants. It was terminated early, so we do not have full results on safety or effectiveness.

What this could mean

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

Active substance
BI 3720931 (inhaled gene therapy)
What this could lead to
If it works, this could provide a new treatment option for people with cystic fibrosis who cannot take current modulator drugs.
What could go wrong
This was a very early, small trial (only 5 participants) and was terminated, so results are limited. Gene therapies can have unexpected side effects and may not improve lung function.

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 CYSTIC FIBROSIS 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

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035, Spain

  • Hôpital Gui de Chauliac

    Montpellier, 34295, France

  • Osp. Pediatrico Bambin Gesù

    Roma, 00165, Italy

  • Royal Brompton Hospital

    London, SW3 6JY, United Kingdom

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX, Netherlands

More trials for these conditions

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