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

NCT05010577 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: BMX-04-001
• Study Type: interventional
• Target: 43
• Locations: 5 countries
• Sites: 28

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.

Conditions

Chronic Pseudomonas Aeruginosa Infection; Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability

  Incidence of treatment emergent adverse events following single and multiple doses of BX004-A administered by inhalation

  6 months

Other Outcomes (1)

• PsA burden in sputum at various timepoints

  1 month

Study Arms (2)

BX004-A

EXPERIMENTAL

Participants will be randomized to receive standard dose of nebulized bacteriophage

Drug: BX004-A

Placebo

PLACEBO COMPARATOR

Participants will be randomized to receive nebulized placebo

Drug: Placebo

Interventions

BX004-A DRUG

Combination of nebulized bacteriophages targeting Pseudomonas aeruginosa

Also known as: Bacteriophage

BX004-A

Placebo DRUG

Nebulized placebo

Also known as: Vehicle buffer

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• BiomX, Inc.: lead

Study Sites (28)

The University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

Providence Alaska Medical Center

Anchorage, Alaska, 99508, United States

Location

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States

Location

Central Florida Pulmonary Group

Orlando, Florida, 32803, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02130, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

University Hospitals Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Baylor College of Medicine, Texas Children Clinic

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

University Hospital in Motol

Prague, 15006, Czechia

Location

Rambam Health Care Campus (RHCC) - Ruth Rappaport Children's Hospital

Haifa, 3109601, Israel

Location

Carmel Medical Center

Haifa, 3436212, Israel

Location

Hadassah University Medical Center

Jerusalem, 91999, Israel

Location

Schneider Children's Medical Center of Israel

Petach Tikvah, 4920235, Israel

Location

The Chaim Sheba Medical Center

Ramat Gan, 52621, Israel

Location

Amsterdam Medical Center

Amsterdam, 1105AZ, Netherlands

Location

University Medical Center Utrecht

Utrecht, 3584EA, Netherlands

Location

Hospital Clinico Universitario Virgen de la Arrixaca

El Palmar, Murcia, 30120, Spain

Location

Vall d'Hebron Barcelona Hospital Campus

Barcelona, 08035, Spain

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Urania Rappo, MD

  BiomX, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 11, 2021
• First Posted: August 18, 2021
• Study Start: June 21, 2022
• Primary Completion: March 10, 2024
• Study Completion: March 10, 2024
• Last Updated: July 18, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

IL (5); US (18); ES (2); CZ (1); NL (2)