Chronic Pseudomonas Aeruginosa Infection, Cystic Fibrosis (CF)
Conditions
Brief summary
The goal of this Phase 2b clinical trial is to see if nebulized phage (BX004) can treat chronic Pseudomonas aeruginosa (PsA) lung infection in CF subjects. The primary goal is to see if 8 weeks of twice daily BX004 can reduce the amount of PsA in the sputum compared to placebo (on top of background CF therapy).
Detailed description
This is a randomized, double-blind, placebo-controlled, multicenter study to evaluate BX004 in CF subjects with chronic PsA pulmonary infection. The main purpose of the study is to evaluate whether BX004 reduces the PsA burden in the sputum of CF subjects with chronic PsA pulmonary infection. Secondary endpoints are to see how well BX004 works in improving lung function and quality of life, reducing the amount of PsA in the sputum, getting negative sputum cultures for PsA, and safety and tolerability. Clinically stable CF subjects with a confirmed diagnosis of CF and chronic PsA pulmonary infection will be enrolled. Subjects will be included in a 6-month post-dose safety follow-up. A Data Safety Monitoring Board of the CF Foundation will monitor safety.
Interventions
BIOLOGICALBX004
Bacteriophage
OTHERPlacebo
Placebo
Sponsors
BiomX Ltd
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 inhaled antibiotics (cycling or continuous regimen) or no inhaled antibiotics * Age ≥ 18 years * FEV1 40%-80% 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 or Day 1 * Detection of Burkholderia cenocepacia from respiratory tract within 1 year prior to Screening or from Screening culture * Currently receiving systemic treatment for allergic bronchopulmonary aspergillosis * Currently receiving treatment for active infection with non-tuberculous mycobacteria or prior detection of Mycobacterium abscessus in 12 months prior to Screening * History of severe neutropenia * History of lung transplant * History of solid organ transplant * Acquired or primary immunodeficiency syndrome * Initiation or change in type of CFTR modulator less than 3 months prior to Screening * Pregnant or breastfeeding female
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in sputum Pseudomonas aeruginosa (PsA) burden at 8 weeks (EOT) | 8 weeks | Change from baseline in PsA colony-forming units (CFU) per g of sputum |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in CFQ-R respiratory domain | until 6 months after last dose of study drug | Cystic Fibrosis Questionnaire - Revised (CFQ-R) respiratory domain: change at D29, D43, D57 (EOT), D85, 3 months post-dose, and 6 months post-dose (range 0-100; higher score=better outcome) |
| Change from Baseline in CFRSD-CRISS | until 6 months after last dose of study drug | Cystic Fibrosis Respiratory Symptom Diary and Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) weekly changes through D14 (daily completion), weekly changes from D21 through D85, and at 3 months post-dose, and 6 months post-dose (range 0-100; higher score=worse outcome) |
| Change in lung function at D8, D29, D43, D57 (EOT), D85, 3 months post-dose, and 6 months post-dose | from Day 8 until 6 months after last dose (end of study) | Change from baseline in % predicted FEV1 |
| Efficacy of BX004 on obtaining negative sputum cultures for PsA | until 6 months after last dose of study drug | Proportion of subjects with negative sputum cultures for PsA, time to negative sputum cultures, durability of negative sputum cultures |
| Incidence of treatment-emergent adverse events [safety and tolerability] | until 6 months after last dose of study drug | Incidence of treatment-emergent adverse events |
| Change in sputum PsA burden | until 6 months after last dose of study drug | Change from baseline in sputum PsA CFU/g at D8, D29, D43, D85, 3 months post-dose and 6 months post-dose |
Countries
United States
Contacts
Primary ContactUrania Rappo, MD
Outcome results
None listed