Clinical trial NCT06998043: Study With Phage for CF Subjects With Pseudomonas Lung Infection
Official title: A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects With Chronic Pseudomonas Aeruginosa (PsA) Pulmonary Infection
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Chronic Pseudomonas Aeruginosa Infection
Cystic Fibrosis (CF)
Lead sponsor: BiomX, Inc.
Sponsor class: INDUSTRY
First posted: 2025-05-31
Enrollment: 63
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Change in sputum Pseudomonas aeruginosa (PsA) burden at 8 weeks (EOT)
timeFrame: 8 weeks
Secondary outcomes:
measure: Change in lung function at D8, D29, D43, D57 (EOT), D85, 3 months post-dose, and 6 months post-dose
timeFrame: from Day 8 until 6 months after last dose (end of study)
measure: Change from Baseline in CFQ-R respiratory domain
timeFrame: until 6 months after last dose of study drug
measure: Change from Baseline in CFRSD-CRISS
timeFrame: until 6 months after last dose of study drug
measure: Change in sputum PsA burden
timeFrame: until 6 months after last dose of study drug
measure: Efficacy of BX004 on obtaining negative sputum cultures for PsA
timeFrame: until 6 months after last dose of study drug
measure: Incidence of treatment-emergent adverse events [safety and tolerability]
timeFrame: until 6 months after last dose of study drug
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Anchorage
country: United States
facility: Providence Alaska Medical Center
state: Alaska
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Hollywood
country: United States
facility: Joe DiMaggio Children's Health
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group
state: Florida
city: Pensacola
country: United States
facility: Avanza Medical Center
state: Florida
city: Hawthorne
country: United States
facility: New York Medical College
state: New York
city: New York
country: United States
facility: Northwell Health
state: New York
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: BiomX, Inc.
name: Urania Rappo, MD
role: STUDY_DIRECTOR
References: