First human test of inhaled gene therapy for cystic fibrosis halted early

NCT ID NCT06515002

Summary

This early-stage study tested a single inhaled dose of an experimental gene therapy called BI 3720931 in adults with cystic fibrosis who cannot use standard CFTR modulator drugs. The goal was to see if the treatment was safe and if it could improve lung function. The trial was terminated after enrolling only 5 participants, and its long-term effects remain unknown.

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Contacts and locations

Locations

  • HOP Gui de Chauliac

    Montpellier, 34295, France

  • Hospital Universitari Vall D Hebron

    Barcelona, 08035, Spain

  • Osp. Pediatrico Bambin Gesù

    Roma, 00165, Italy

  • Royal Brompton Hospital

    London, SW3 6JY, United Kingdom

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX, Netherlands

Conditions

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