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

Sponsor

BiomX, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05010577

Collaborator

(none)

Enrollment

Locations

Arms

13.3

Anticipated Duration (Months)

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Chronic Pseudomonas Aeruginosa Infection Cystic Fibrosis	Drug: BX004-A Drug: Placebo	Phase 1/Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

32 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Jun 21, 2022

Anticipated Primary Completion Date :

Mar 1, 2023

Anticipated Study Completion Date :

Aug 1, 2023

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Safety and tolerability [6 months]
Incidence of treatment emergent adverse events following single and multiple doses of BX004-A administered by inhalation

Other Outcome Measures

PsA burden in sputum at various timepoints [1 month]
Change in PsA colony-forming units (CFU) per gram of sputum

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 2 years 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Joe DiMaggio Children's Hospital	Hollywood	Florida	United States	33021
2	Central Florida Pulmonary Group	Orlando	Florida	United States	32803
3	Boston Children's Hospital	Boston	Massachusetts	United States	02130
4	New York Medical College	Valhalla	New York	United States	10595
5	University Hospitals Rainbow Babies and Children's Hospital	Cleveland	Ohio	United States	44106
6	Penn State Health Milton S. Hershey Medical Center	Hershey	Pennsylvania	United States	17033
7	Seattle Children's Hospital	Seattle	Washington	United States	98105
8	Hadassah University Medical Center	Jerusalem		Israel	91999

Sponsors and Collaborators

BiomX, Inc.

Investigators

Study Director: Xilla Ussery, MD, BiomX, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

BiomX, Inc.

ClinicalTrials.gov Identifier:

NCT05010577

Other Study ID Numbers:

BMX-04-001

First Posted:

Aug 18, 2021

Last Update Posted:

Jun 30, 2022

Last Verified:

Jun 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Infections

Communicable Diseases

Pseudomonas Infections

Cystic Fibrosis

Fibrosis

Study Results

No Results Posted as of Jun 30, 2022