Study With Phage for CF Subjects With Pseudomonas Lung Infection

Phase 2RecruitingNCT06998043

BiomX Ltd63 enrolled

Overview

The goal of this Phase 2b clinical trial is to see if nebulized phage (BX004) can treat chronic Pseudomonas aeruginosa (PsA) lung infection in CF subjects. The primary goal is to see if 8 weeks of twice daily BX004 can reduce the amount of PsA in the sputum compared to placebo (on top of background CF therapy).

This is a randomized, double-blind, placebo-controlled, multicenter study to evaluate BX004 in CF subjects with chronic PsA pulmonary infection. The main purpose of the study is to evaluate whether BX004 reduces the PsA burden in the sputum of CF subjects with chronic PsA pulmonary infection. Secondary endpoints are to see how well BX004 works in improving lung function and quality of life, reducing the amount of PsA in the sputum, getting negative sputum cultures for PsA, and safety and tolerability. Clinically stable CF subjects with a confirmed diagnosis of CF and chronic PsA pulmonary infection will be enrolled. Subjects will be included in a 6-month post-dose safety follow-up. A Data Safety Monitoring Board of the CF Foundation will monitor safety.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Chronic Pseudomonas Aeruginosa Infection Cystic Fibrosis (CF)

Interventions

BX004BIOLOGICAL

Bacteriophage

PlaceboOTHER

Placebo

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Cystic fibrosis patients with chronic Pseudomonas aeruginosa pulmonary infection receiving standard of care inhaled antibiotics (cycling or continuous regimen) or no inhaled antibiotics * Age ≥ 18 years * FEV1 40%-80% 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 or Day 1 * Detection of Burkholderia cenocepacia from respiratory tract within 1 year prior to Screening or from Screening culture * Currently receiving systemic treatment for allergic bronchopulmonary aspergillosis * Currently receiving treatment for active infection with non-tuberculous mycobacteria or prior detection of Mycobacterium abscessus in 12 months prior to Screening * History of severe neutropenia * History of lung transplant * History of solid organ transplant * Acquired or primary immunodeficiency syndrome * Initiation or change in type of CFTR modulator less than 3 months prior to Screening * Pregnant or breastfeeding female

Locations (14)

University of Alabama at Birmingham

Birmingham, Alabama, United States

RECRUITING

Providence Alaska Medical Center

Anchorage, Alaska, United States

Outcomes

Primary Outcomes

Change in sputum Pseudomonas aeruginosa (PsA) burden at 8 weeks (EOT)

Change from baseline in PsA colony-forming units (CFU) per g of sputum

Time frame: 8 weeks

Secondary Outcomes

Change in lung function at D8, D29, D43, D57 (EOT), D85, 3 months post-dose, and 6 months post-dose

Change from baseline in % predicted FEV1

Time frame: from Day 8 until 6 months after last dose (end of study)

Change from Baseline in CFQ-R respiratory domain

Cystic Fibrosis Questionnaire - Revised (CFQ-R) respiratory domain: change at D29, D43, D57 (EOT), D85, 3 months post-dose, and 6 months post-dose (range 0-100; higher score=better outcome)

Time frame: until 6 months after last dose of study drug

Change from Baseline in CFRSD-CRISS

Cystic Fibrosis Respiratory Symptom Diary and Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) weekly changes through D14 (daily completion), weekly changes from D21 through D85, and at 3 months post-dose, and 6 months post-dose (range 0-100; higher score=worse outcome)

Time frame: until 6 months after last dose of study drug

Change in sputum PsA burden

Change from baseline in sputum PsA CFU/g at D8, D29, D43, D85, 3 months post-dose and 6 months post-dose

Time frame: until 6 months after last dose of study drug

Efficacy of BX004 on obtaining negative sputum cultures for PsA

Proportion of subjects with negative sputum cultures for PsA, time to negative sputum cultures, durability of negative sputum cultures

Time frame: until 6 months after last dose of study drug

Central Contacts

Urania Rappo, MD

CONTACT

1-617-256-2625 uraniar@biomx.com

Data from ClinicalTrials.gov

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