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

Phase 1
Active, not recruiting
Conditions
Chronic Pseudomonas Aeruginosa Infection
Cystic Fibrosis
Interventions
Drug: Placebo
Registration Number
NCT05010577
Lead Sponsor
BiomX, Inc.
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.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
32
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 & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants will be randomized to receive nebulized placebo
BX004-ABX004-AParticipants will be randomized to receive standard dose of nebulized bacteriophage
Primary Outcome Measures
NameTimeMethod
Safety and tolerability6 months

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

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (28)

Boston Children's Hospital

🇺🇸

Boston, Massachusetts, United States

University Hospitals Rainbow Babies and Children's Hospital

🇺🇸

Cleveland, Ohio, United States

Nationwide Children's Hospital

🇺🇸

Columbus, Ohio, United States

Baylor College of Medicine, Texas Children Clinic

🇺🇸

Houston, Texas, United States

Seattle Children's Hospital

🇺🇸

Seattle, Washington, United States

University Hospital in Motol

🇨🇿

Prague, Czechia

Providence Alaska Medical Center

🇺🇸

Anchorage, Alaska, United States

University of Arkansas for Medical Sciences

🇺🇸

Little Rock, Arkansas, United States

Amsterdam Medical Center

🇳🇱

Amsterdam, Netherlands

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

🇮🇱

Haifa, Israel

Hadassah University Medical Center

🇮🇱

Jerusalem, Israel

New York Medical College

🇺🇸

Valhalla, New York, United States

The Chaim Sheba Medical Center

🇮🇱

Ramat Gan, Israel

Hospital Clinico Universitario Virgen de la Arrixaca

🇪🇸

El Palmar, Murcia, Spain

University of Florida

🇺🇸

Gainesville, Florida, United States

Children's National Medical Center

🇺🇸

Washington, District of Columbia, United States

Penn State Health Milton S. Hershey Medical Center

🇺🇸

Hershey, Pennsylvania, United States

Carmel Medical Center

🇮🇱

Haifa, Israel

Schneider Children's Medical Center of Israel

🇮🇱

Petach Tikvah, Israel

Vall d'Hebron Barcelona Hospital Campus

🇪🇸

Barcelona, Spain

Joe DiMaggio Children's Hospital

🇺🇸

Hollywood, Florida, United States

The University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Central Florida Pulmonary Group

🇺🇸

Orlando, Florida, United States

Yale University

🇺🇸

New Haven, Connecticut, United States

Cincinnati Children's Hospital

🇺🇸

Cincinnati, Ohio, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

University of Utah

🇺🇸

Salt Lake City, Utah, United States

University Medical Center Utrecht

🇳🇱

Utrecht, Netherlands

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