Clinical trial NCT05010577: Nebulized Bacteriophage Therapy in Cystic Fibrosis Patients With Chronic Pseudomonas Aeruginosa Pulmonary Infection
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Chronic Pseudomonas Aeruginosa Infection
Cystic Fibrosis
Lead sponsor: BiomX, Inc.
Sponsor class: INDUSTRY
First posted: 2021-08-18
Enrollment: 32
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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Safety and tolerability
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
measure: PsA burden in sputum at various timepoints
timeFrame: 1 month
Locations:
city: Birmingham
country: United States
facility: The 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: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Hollywood
country: United States
facility: Joe DiMaggio Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group
state: Florida
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Valhalla
country: United States
facility: New York Medical College
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Rainbow Babies and Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Houston
country: United States
facility: Baylor College of Medicine, Texas Children Clinic
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Prague
country: Czechia
facility: University Hospital in Motol
city: Haifa
country: Israel
facility: Rambam Health Care Campus (RHCC) - Ruth Rappaport Children's Hospital
city: Haifa
country: Israel
facility: Carmel Medical Center
city: Jerusalem
country: Israel
facility: Hadassah University Medical Center
city: Petach Tikvah
country: Israel
facility: Schneider Children's Medical Center of Israel
city: Ramat Gan
country: Israel
facility: The Chaim Sheba Medical Center
city: Amsterdam
country: Netherlands
facility: Amsterdam Medical Center
city: Utrecht
country: Netherlands
facility: University Medical Center Utrecht
city: El Palmar
country: Spain
facility: Hospital Clinico Universitario Virgen de la Arrixaca
state: Murcia
city: Barcelona
country: Spain
facility: Vall d'Hebron Barcelona Hospital Campus
Study officials:
affiliation: BiomX, Inc.
name: Urania Rappo, MD
role: STUDY_DIRECTOR
References: