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Gene Therapy Trial Report

Summary

A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)


NCTID NCT06515002 (View at clinicaltrials.gov)
Description
Development Status 🔄 Inactive
Indication Cystic Fibrosis
Disease Ontology Term DOID:1485
Compound Name BI 3720931
Compound Description rSIV.F/HN-hCEF-soCFTR2
Sponsor Boehringer Ingelheim
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 5 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant CFTR
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Inhalational
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type rSIV.F/HN
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-07-18
Completion Date 2025-10-30
Last Update 2026-02-19

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key inclusion criteria: * Cystic fibrosis (CF)-pulmonary phenotype and a confirmed diagnosis of CF: * Positive sweat chloride ≥60 mmol/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 cystic fibrosis transmembrane conductance regulator modulator therapy (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 * Forced expiratory volume in 1 second, percent of predicted value (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 Further inclusion criteria apply. Key exclusion criteria: * Trial 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 5
Locations Netherlands,Italy,United Kingdom,France,Spain

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Sponsor terminated the study in February 2026 after initial data readout

Resources/Links