2023-503281-23-00
Recruiting
Phase 1/2
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)
Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A.6 sites in 4 countries12 target enrollmentStarted: June 17, 2024Last updated:
Overview
- Phase
- Phase 1/2
- Status
- Recruiting
- Sponsor
- Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A.
- Enrollment
- 12
- Locations
- 6
- Primary Endpoint
- Phase I: Occurrence of any drug-related, treatment-emergent AE up to Week 24 after drug administration
Overview
Brief Summary
"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. "
Study Design
- Allocation
- Randomized
- Primary Purpose
- Phase II: Follow up
- Masking
- Double (Investigator, Subject, Analyst, Monitor, Carer)
Eligibility Criteria
- Ages
- 18 years to 65+ years (18-64 Years, 65+ Years)
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
- •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 Criteria
- •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.
Outcomes
Primary Outcomes
Phase I: Occurrence of any drug-related, treatment-emergent AE up to Week 24 after drug administration
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 II: Absolute change from baseline in FEV1pp at Week 8 after drug administration
Secondary Outcomes
- 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
Investigators
CT Disclosure & Data Transparency
Scientific
Boehringer Ingelheim International GmbH
Study Sites (6)
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