Testing a new medication for lung function in cystic fibrosis patients

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 (Lenticlair 1)

This study is testing a new medication to see if it can help improve lung function in adults with cystic fibrosis who can't use current treatments.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Boehringer Ingelheim (Industry-sponsored)
• Locations: 10 sites (Montpellier and 9 other locations)
• Trial ID: NCT06515002 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and effectiveness of a medication called BI 3720931 in improving lung function for adults with cystic fibrosis who are not eligible for existing CFTR modulator therapies. The study is divided into two phases: Phase 1 assesses different doses of the medication to determine tolerance, while Phase 2 compares the effects of BI 3720931 against a placebo. Participants will receive a single dose via inhalation and will be monitored for lung function improvements. This is the first time BI 3720931 is being tested in humans.

Who should consider this trial

Good fit: Ideal candidates include adult men and women with cystic fibrosis who are not eligible for CFTR modulator therapy and have a specific lung function range.

Not a fit: Patients who are eligible for CFTR modulator therapy or have severe lung function impairment may not benefit from this study.

Why it matters

Potential benefit: If successful, this medication could provide a new treatment option for cystic fibrosis patients who currently have limited alternatives.

How similar studies have performed: While this approach is novel in testing BI 3720931, similar studies targeting lung function improvement in cystic fibrosis have shown promise.

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.

Where this trial is running

Montpellier and 9 other locations

• HOP Gui de Chauliac — Montpellier, France (Recruiting)
• HOP Necker — Paris, France (Not_yet_recruiting)
• Istituto G. Gaslini — Genova, Italy (Not_yet_recruiting)
• Osp. Pediatrico Bambin Gesù — Roma, Italy (Recruiting)
• Universitair Medisch Centrum Utrecht — Utrecht, Netherlands (Recruiting)
• Hospital Vall d'Hebron — Barcelona, Spain (Recruiting)
• University Hospital Llandough — Cardiff, United Kingdom (Not_yet_recruiting)
• Queen Elizabeth University Hospital — Glasgow, United Kingdom (Not_yet_recruiting)
• Royal Brompton Hospital — London, United Kingdom (Recruiting)
• Wythenshawe Hospital — Manchester, United Kingdom (Not_yet_recruiting)

Study contacts

• Study coordinator: Boehringer Ingelheim

Conditions Cystic Fibrosis