Gene therapy trial for cystic fibrosis halted early
NCT ID NCT06515002
First seen Jun 25, 2026 · Last updated Jun 25, 2026
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Hospital Universitari Vall d'Hebron
Barcelona, 08035, Spain
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Hôpital Gui de Chauliac
Montpellier, 34295, France
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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
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.
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.