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Lenticlair™ 1: A Phase 1/2 trial evaluating the safety, tolerability and efficacy of an inhaled F/HN-pseudotyped lentiviral vector for CF gene therapy in people with CF ineligible for CFTR modulators 

J C Davies(1,2) M A Mall(3) D Polineni(4) S H Donaldson(5) I Fajac(6,7) R Jain(8) B K Rubin(9) A C Boyd(10) D R Gill(11) U Griesenbach(1) S C Hyde(11) G McLachlan(12) M Avis(13) C Diefenbach(14) R Sigmund(14) W Seibold(15) A Gupta(15) E WFW Alton(1,2)

1:National Heart and Lung Institute, Imperial College London, London, UK; 2:Royal Brompton Hospital, part of Guy's & St Thomas' NHS Foundation Trust, London, UK; 3:Department of Pediatrics Respiratory Medicine, Immunology and Critical Care Medicine, Charité-Universitätsmedizin Berlin, Berlin, Germany; 4:Department of Pediatrics, Division of Allergy & Pulmonary Medicine, Washington University School of Medicine in St. Louis, St. Louis, MO, USA; 5:Department of Medicine, Division of Pulmonary Diseases and Critical Care Medicine, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; 6:Université Paris Cité, Paris, France; 7:AP-HP, Hôpital Cochin, Service de Physiologie et Explorations Fonctionnelles, Paris, France; 8:Department of Internal Medicine, University of Texas Southwestern Medical Center Dallas, TX, USA; 9:Department of Pediatrics, Virginia Commonwealth University School of Medicine, Richmond, VA, USA; 10:Centre for Genomic & Experimental Medicine Institute of Genetics & Cancer, Western General Hospital, University of Edinburgh, Edinburgh, UK; 11:Gene Medicine Group, Nuffield Division of Clinical Laboratory Science, Radcliffe Department of Medicine, University of Oxford, Oxford, UK; 12:The Roslin Institute, University of Edinburgh, Edinburgh, UK; 13:Boehringer Ingelheim B.V., Amsterdam, Netherlands; 14:Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach an der Riss, Germany; 15:Boehringer Ingelheim International GmbH, Biberach an der Riss, Germany

This first-in-human Phase 1/2 trial will evaluate the safety and efficacy of BI 3720931, a third-generation lentiviral vector, in people with CF (pwCF) who are genetically ineligible for CF transmembrane conductance regulator modulator therapy (CFTRmt). 

pwCF: ≥18 years; percent predicted forced expiratory volume in 1 second (ppFEV1) 50–100% at screening; no recent exacerbations; ineligible for CFTRmt; naïve to prior GT, or prior exposure to non-viral/viral GT with drug-free intervals of 6/24 months. Phase 1: open-label dose-escalation trial (n=9) of single low/medium/high-dose nebulised BI 3720931 (3/group) plus standard of care (SoC) with 24 weeks’ follow-up (FU). Primary endpoint: drug-related adverse events (AEs) within 24 weeks. Secondary endpoints: change from baseline ppFEV₁ ≥5% within 8 weeks; absolute change from baseline in ppFEV₁ at Week 24; dose-limiting toxicity up to Week 24. Bronchoscopy will be performed to quantify CFTR expression. Interim data from ≥8 weeks post-dose will inform Phase 2 dose selection. Phase 2 (n=27): randomised, double-blind, placebo-controlled, dose-expansion trial of single-dose nebulised BI 3720931 (2 dose levels), or placebo plus SoC (1:1:1) with 24 weeks’ FU. Primary endpoint: absolute change from baseline in ppFEV₁ at Week 8; secondary endpoints: absolute change from baseline in ppFEV₁ at Week 24, and occurrence of serious and drug-related AEs up to Week 24. 

The trial is estimated to begin in Q2 2024. 

The trial will evaluate safety and efficacy of BI 3720931 over 24 weeks in pwCF who are ineligible for CFTRmt.  

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