A study to test how well BI 3720931 is tolerated and whether it improves lung function in people with cystic fibrosis (LenticlairTM 1)

Trial details

Who runs it

Boehringer Ingelheim International GmbH

Trial code

EU 2023-503281-23-00

Record updated

2025. július 22.

Original description

"Phase I: To investigate safety and tolerability of a single inhaled dose of BI 3720931 based on number of trial participants with at least one drug-related, treatment emergent AE up to Week 24 after dosing. Phase II: To demonstrate superiority of the higher BI 3720931 dose over PBO on the primary endpoint, the absolute change from baseline in FEV1pp at Week 8. If superiority for the high dose is established, superiority of the lower dose versus PBO will be tested in a hierarchical manner on the primary endpoint. "Read full original descriptionShow less

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)

"Phase I: To investigate safety and tolerability of a single inhaled dose of BI 3720931 based on number of trial participants with at least one drug-related, treatment emergent AE up to Week 24 after dosing. Phase II: To demonstrate superiority of the higher BI 3720931 dose over PBO on the primary endpoint, the absolute change from baseline in FEV1pp at Week 8. If superiority for the high dose is established, superiority of the lower dose versus PBO will be tested in a hierarchical manner on the primary endpoint. "

Inclusion: Male or female of non-childbearing potential trial participants with a CF-pulmonary phenotype and a confirmed diagnosis of CF: - Positive sweat chloride ≥60 mEq/L by pilocarpine iontophoresis OR - Genotype with 2 identifiable CF-causing mutations accompanied by one or more clinical features if sweat chloride testing is between 30 and 59 mmol/L; Trial participants who are not eligible for treatment with CFTRmt due to their genotype with 2 identified CFTR-mutations (including Class I CFTR gene mutations) and are also not expected to become eligible during the trial according to investigator´s opinion; Trial participants able to perform acceptable spirometric maneuvers according to American Thoracic Society/European Respiratory Society 2019 standards; FEV1pp ≥50% and ≤100% of predicted normal at Visit 1. Predicted value based on Global Lung Initiative lung function reference equations; Stable CF disease with no pulmonary exacerbation 4 weeks prior to the screening visit and during the screening period and stable drug- and non-drug therapy for CF in the 4 weeks prior to dosing; Trial participants either naïve to prior gene therapy or exposed to prior viral or non-viral gene therapies for cystic fibrosis. If prior exposure to gene therapy for CF exists, then the following applies as per investigator judgement: a. No apparent residual side effects associated with prior gene therapy as per investigator assessment, and b. Drug-free interval of -- 6 months after last dose of prior non-viral gene therapy -- 24 months after last dose of prior viral gene therapy; Further inclusion criteria apply.

Exclusion: Trial participants with ongoing or planned CFTRmt, or participants not eligible for CFTRmt based on contraindications (e.g. liver failure) or who needed to withdraw CFTRmt due to intolerability are not appropriate candidates for this Phase I/II trial; Trial participants requiring chronic use of systemic corticosteroids or immunosuppressants to treat another condition; Further exclusion criteria apply.

EU CTIS 2023-503281-23-00