BX004 for Cystic Fibrosis

Recruiting at 13 trial locations

Overseen By Urania Rappo, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: BiomX Ltd

Must be taking: Inhaled antibiotics

Must not be taking: Systemic antifungals

Disqualifiers: Lung transplant, Immunodeficiency, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

63 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called BX004, a nebulized phage therapy, to determine its effectiveness for people with cystic fibrosis who have a chronic lung infection caused by Pseudomonas aeruginosa. The goal is to assess whether using BX004 twice daily for eight weeks can reduce the bacteria in the lungs. Participants will receive either BX004 or a placebo, alongside their usual cystic fibrosis care. The trial seeks individuals with cystic fibrosis who have stable lung disease and this specific chronic lung infection. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in cystic fibrosis care.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop your current medications. However, it mentions that participants can be on standard inhaled antibiotics or not, so it seems you may continue your current CF treatments.

Is there any evidence suggesting that BX004 is likely to be safe for humans?

Research shows that BX004, a treatment being tested for cystic fibrosis (CF), has shown promise in earlier studies. In a previous study with adults who have CF, BX004 was generally well-tolerated, meaning most people did not experience serious side effects, indicating good safety.

The current trial is in an early phase, focusing mainly on safety and tolerability. BX004 reaching this stage suggests some confidence in its safety from earlier tests. This trial primarily aims to see if BX004 can reduce lung infections caused by Pseudomonas aeruginosa, a type of bacteria, in people with CF. While ongoing studies will provide more information, early results are encouraging for potential participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for cystic fibrosis, which typically include antibiotics and medications to thin mucus, BX004 is unique because it uses a bacteriophage approach. Bacteriophages are viruses that specifically target and kill bacteria, offering a new way to combat the bacterial infections common in cystic fibrosis patients. This innovative mechanism of action could potentially reduce antibiotic resistance and provide a more effective treatment. Researchers are excited about BX004 because it represents a novel strategy that might improve outcomes for patients in a way that traditional treatments haven’t been able to achieve.

What evidence suggests that BX004 might be an effective treatment for cystic fibrosis?

Research has shown that BX004, a mist-based treatment using viruses that target bacteria, may help treat lung infections caused by Pseudomonas aeruginosa in people with cystic fibrosis. In earlier studies, BX004 reduced the number of harmful bacteria by 500 times without the bacteria becoming resistant, significantly decreasing bacteria levels. In this trial, participants will receive either the standard dose of nebulized BX004 or a nebulized placebo, determined randomly. The therapy is delivered directly to the lungs using a nebulizer, providing a focused treatment. Overall, promising evidence suggests that BX004 could effectively reduce infections in cystic fibrosis patients. ^{1 3 6 7 8}

Who Is on the Research Team?

Urania Rappo, MD

Principal Investigator

BiomX, Inc.

Are You a Good Fit for This Trial?

This trial is for individuals with Cystic Fibrosis (CF) who have a chronic lung infection caused by Pseudomonas aeruginosa. Participants should be on stable CF therapy and able to provide sputum samples. Specific eligibility criteria are not provided, but typically include age range, disease severity, and absence of conditions that could interfere with the study.

Inclusion Criteria

I am 18 years old or older.

FEV1 40%-80% predicted

Willing and able to provide adequate sputum samples, using any method (spontaneously expectorated, induced, from home or clinic) at designated study visits.

Exclusion Criteria

Known hypersensitivity to bacteriophages or excipients in the formulation

Detection of Burkholderia cenocepacia from respiratory tract within 1 year prior to Screening or from Screening culture

History of lung transplant

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive nebulized phage (BX004) or placebo twice daily for 8 weeks

8 weeks

Multiple visits for dosing and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Regular follow-up visits

What Are the Treatments Tested in This Trial?

Interventions

• BX004

Trial Overview

The trial is testing BX004, a nebulized phage therapy designed to treat Pseudomonas aeruginosa infections in the lungs of CF patients. Over 8 weeks, participants will inhale BX004 or a placebo twice daily to see if there's a reduction in bacterial levels in their sputum.

How Is the Trial Designed?

Group I: BX004 Experimental Treatment 1 Intervention

Participants will be randomized to receive standard dose of nebulized bacteriophage

Group II: Placebo Placebo Group 1 Intervention

Participants will be randomized to receive nebulized placebo

Who Is Running the Clinical Trial?

BiomX Ltd

Lead Sponsor

Citations

1.

nature.com

nature.com/articles/s41467-025-60598-4

Phage therapy with nebulized cocktail BX004-A for chronic ...

Taken together, these data demonstrate the favorable safety profile, feasibility of lower respiratory tract delivery, and preliminary ...

2.

ir.biomx.com

ir.biomx.com/news-releases/news-release-details/biomx-provides-update-bx004-phase-2b-trial-treatment-patients

BiomX Provides Update on BX004 Phase 2b Trial for the ...

U.S. FDA has placed a clinical hold on the Phase 2b study as it reviews data submitted by BiomX on third-party nebulizer used to deliver ...

3.

pharmatimes.com

pharmatimes.com/news/biomx-phage-therapy-shows-potent-effect-in-cystic-fibrosis-trial/

BiomX phage therapy shows potent effect in cystic fibrosis ...

BX004 achieves 500-fold bacterial reduction with no resistance in phase 1b/2a study.

4.

clinicaltrialsarena.com

clinicaltrialsarena.com/news/biomx-cystic-fibrosis-treatment/

BiomX doses first subject in trial of cystic fibrosis treatment

The study's design includes a range of efficacy endpoints, such as a reduction in bacterial burden, lung function improvement, and improvements ...

5.

cysticfibrosisnewstoday.com

cysticfibrosisnewstoday.com/news/trial-cf-treatment-bx004-nears-top-line-results-europe/

Trial for CF treatment BX004 nears top-line results in Europe

Top-line European results from a trial testing BX004, an experimental CF treatment, are expected in 2026 as the U.S. trial remains on hold.

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05010577

Study Details | NCT05010577 | Nebulized Bacteriophage ...

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 ...

7.

rarediseaseadvisor.com

rarediseaseadvisor.com/news/biomx-announces-positive-topline-data-bx004-cf-treatment/

BiomX Announces Positive Topline Data for BX004 to ...

According to topline data, BX004 lead to clinically significant improvement in pulmonary function compared with placebo, as evidenced by ...

8.

clinicaltrials.ucsf.edu

clinicaltrials.ucsf.edu/trial/NCT06998043

Phage for CF Subjects With Pseudomonas Lung Infection

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.