Nebulized Bacteriophage Therapy in Cystic Fibrosis Patients With Chronic Pseudomonas Aeruginosa Pulmonary Infection

A Phase 1b/2a, 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

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.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis; Chronic Pseudomonas Aeruginosa Infection
• Intervention / Treatment: Drug: Placebo; Drug: BX004-A

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.

• Study Type: Interventional
• Enrollment (Actual): 43
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Czechia
  • Prague, Czechia, 15006
    • University Hospital in Motol
• Israel
  • Haifa, Israel, 3436212
    • Carmel Medical Center
  • Haifa, Israel, 3109601
    • Rambam Health Care Campus (RHCC) - Ruth Rappaport Children's Hospital
  • Jerusalem, Israel, 91999
    • Hadassah University Medical Center
  • Petach Tikvah, Israel, 4920235
    • Schneider Children's Medical Center of Israel
  • Ramat Gan, Israel, 52621
    • The Chaim Sheba Medical Center
• Netherlands
  • Amsterdam, Netherlands, 1105AZ
    • Amsterdam Medical Center
  • Utrecht, Netherlands, 3584EA
    • University Medical Center Utrecht
• Spain
  • Barcelona, Spain, 08035
    • Vall d'Hebron Barcelona Hospital Campus
  • Murcia
    • El Palmar, Murcia, Spain, 30120
      • Hospital Clinico Universitario Virgen de La Arrixaca
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • The University of Alabama at Birmingham
  • Alaska
    • Anchorage, Alaska, United States, 99508
      • Providence Alaska Medical Center
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas for Medical Sciences
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University
  • District of Columbia
    • Washington, DC, District of Columbia, United States, 20010
      • Children's National Medical Center
  • Florida
    • Gainesville, Florida, United States, 32608
      • University of Florida
    • Hollywood, Florida, United States, 33021
      • Joe DiMaggio Children's Hospital
    • Orlando, Florida, United States, 32803
      • Central Florida Pulmonary Group
  • Massachusetts
    • Boston, Massachusetts, United States, 02130
      • Boston Children's Hospital
  • New York
    • Valhalla, New York, United States, 10595
      • New York Medical College
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Rainbow Babies and Children's Hospital
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Health Milton S. Hershey Medical Center
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Texas
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine, Texas Children Clinic
  • Utah
    • Salt Lake City, Utah, United States, 84132
      • University of Utah
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BX004-A; Participants will be randomized to receive standard dose of nebulized bacteriophage	Drug: BX004-A; Combination of nebulized bacteriophages targeting Pseudomonas aeruginosa; Other Names: Bacteriophage
Placebo Comparator: Placebo; Participants will be randomized to receive nebulized placebo	Drug: Placebo; Nebulized placebo; Other Names: Vehicle buffer

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability	Incidence of treatment emergent adverse events following single and multiple doses of BX004-A administered by inhalation	6 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
PsA burden in sputum at various timepoints	Change in PsA colony-forming units (CFU) per gram of sputum	1 month

Collaborators and Investigators

• Sponsor: BiomX, Inc.
• Investigators: Study Director: Urania Rappo, MD, BiomX, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 21, 2022
• Primary Completion (Actual): March 10, 2024
• Study Completion (Actual): March 10, 2024

Study Registration Dates

• First Submitted: August 11, 2021
• First Submitted That Met QC Criteria: August 11, 2021
• First Posted (Actual): August 18, 2021

Study Record Updates

• Last Update Posted (Actual): July 18, 2025
• Last Update Submitted That Met QC Criteria: July 16, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Bacterial Infections; Bacterial Infections and Mycoses; Gram-Negative Bacterial Infections; Fibrosis; Infections; Communicable Diseases; Cystic Fibrosis; Pseudomonas Infections
• Other Study ID Numbers: BMX-04-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No