A seamless Phase I/II trial with an initial open-label dose escalation part and a subsequent randomised, double-blind, placebo-controlled expansion part to evaluate the safety, tolerability, and efficacy of a single dose of BI 3720931, an inhaled lentiviral vector gene therapy, in adult people with cystic fibrosis who are ineligible for CFTR modulators (LenticlairTM 1)

Cystic fibrosis

Phase I: Occurrence of any drug-related, treatment-emergent AE up to Week 24 after drug administration, Phase II: Absolute change from baseline in FEV1pp at Week 8 after drug administration

Phase I: Occurrence of treatment response defined as change from baseline ≥5% in FEV1pp, comparing the mean of 3 pre-treatment FEV1pp measured in the screening period with the mean of 3 post treatment FEV1pp values at Weeks 4, 6, and 8, Phase I: Absolute change from baseline in FEV1pp at Week 24 after drug administration, Phase I: Occurrence of any DLTs up to Week 24 after drug administration, Phase II: Absolute change from baseline in FEV1pp at Week 24 after drug administration, Phase II: Occurrence of any SAEs up to Week 24 after drug administration, Phase II: Occurrence of any drug-related, treatment-emergent AEs up to Week 24 after drug administration

No related papers found yet.

Papers published before 2007-09-01 may not appear yet. Historical indexing is in progress.

France, Italy, Netherlands, Spain