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

• ClinicalTrials.gov Identifier: NCT05010577
• Recruitment Status: Active, not recruiting
• First Posted: August 18, 2021
• Last Update Posted: October 18, 2023
• Sponsor: BiomX, Inc.
• Information provided by (Responsible Party): BiomX, Inc.

Tracking Information

• First Submitted Date: August 11, 2021
• First Posted Date: August 18, 2021
• Last Update Posted Date: October 18, 2023
• Actual Study Start Date: June 21, 2022
• Estimated Primary Completion Date: October 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 11, 2021)

Safety and tolerability [ Time Frame: 6 months ]

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

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided

Current Other Pre-specified Outcome Measures (submitted: August 11, 2021)

PsA burden in sputum at various timepoints [ Time Frame: 1 month ]

Change in PsA colony-forming units (CFU) per gram of sputum

• Original Other Pre-specified Outcome Measures: Same as current

Descriptive Information

• Brief Title: 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
• 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.
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Chronic Pseudomonas Aeruginosa Infection
• Cystic Fibrosis

Intervention

• Drug: BX004-A
  Combination of nebulized bacteriophages targeting Pseudomonas aeruginosa
  Other Name: Bacteriophage
• Drug: Placebo
  Nebulized placebo
  Other Name: Vehicle buffer

Study Arms

• Experimental: BX004-A
  Participants will be randomized to receive standard dose of nebulized bacteriophage
  Intervention: Drug: BX004-A
• Placebo Comparator: Placebo
  Participants will be randomized to receive nebulized placebo
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: August 11, 2021): 32
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: March 2024
• Estimated Primary Completion Date: October 2023 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Czechia, Israel, Netherlands, Spain, United States

Administrative Information

• NCT Number: NCT05010577
• Other Study ID Numbers: BMX-04-001
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: BiomX, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: BiomX, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Urania Rappo, MD; BiomX, Inc.

• PRS Account: BiomX, Inc.
• Verification Date: October 2023