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.
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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
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Royal Brompton Hospital
London, SW3 6JY, United Kingdom
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Could a single antibiotic be enough for cystic fibrosis lung infections?
- Early use of powerful CF drugs may boost growth in young children
- Hands-On therapy may soothe cystic fibrosis pain without drugs