Chronic Pseudomonas Aeruginosa Infection, Cystic Fibrosis
Conditions
Brief summary
This is a Phase 1b/2a study with the primary objective to determine if BX004-A is safe and tolerable. Exploratory objectives include whether BX004-A reduces sputum Pseudomonas aeruginosa (PsA) bacterial load in CF subjects with chronic PsA pulmonary infection.
Detailed description
This is a randomized, double-blind, placebo-controlled, multicenter study to evaluate the safety and tolerability of BX004-A in CF subjects with chronic PsA pulmonary infection. The study is divided into two parts, a single-ascending and multiple-dose phase (Part 1) and a multiple dose phase (Part 2). Subjects in both parts will be included in a 6-month safety follow-up. A Data Safety Monitoring Board will monitor safety in both parts. The purpose of the study is to evaluate safety and tolerability of BX004-A, and whether BX004-A reduces the PsA burden in the sputum of CF subjects with chronic PsA pulmonary infection. Clinically stable CF subjects with a confirmed diagnosis of CF and chronic PsA pulmonary infection will be enrolled.
Interventions
DRUGBX004-A
Combination of nebulized bacteriophages targeting Pseudomonas aeruginosa
DRUGPlacebo
Nebulized placebo
Sponsors
BiomX, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Cystic fibrosis patients with chronic Pseudomonas aeruginosa pulmonary infection receiving standard of care CF medications * Age ≥ 18 years * FEV1 ≥ 40% predicted * Clinically stable lung disease * Willing and able to provide adequate sputum samples, using any method (spontaneously expectorated, induced, from home or clinic) at designated study visits. Key
Exclusion criteria
* Known hypersensitivity to bacteriophages or excipients in the formulation. * Receipt of prior bacteriophage therapy within the 6 months prior to Screening * Recovery of Burkholderia species from respiratory tract within 1 year prior to screening * Currently receiving treatment for allergic bronchopulmonary aspergillosis * Currently receiving treatment for active infection with non-tuberculous mycobacteria * History of severe neutropenia * History of lung transplant * History of solid organ transplant * Acquired or primary immunodeficiency syndrome * Initiation or change in CF modulator therapy less than 3 months prior to screening * Pregnant or breastfeeding female
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability | 6 months | Incidence of treatment emergent adverse events following single and multiple doses of BX004-A administered by inhalation |
Other
| Measure | Time frame | Description |
|---|---|---|
| PsA burden in sputum at various timepoints | 1 month | Change in PsA colony-forming units (CFU) per gram of sputum |
Countries
Czechia, Israel, Netherlands, Spain, United States
Outcome results
None listed