BX004-A for Cystic Fibrosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Central Florida Pulmonary Group, Orlando, FL
Cystic Fibrosis+5 More
BX004-A - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

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

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Eligible Conditions

  • Cystic Fibrosis
  • Chronic Pseudomonas Aeruginosa Infection

Treatment Effectiveness

Study Objectives

This trial is evaluating whether BX004-A will improve 1 primary outcome and 1 other outcome in patients with Cystic Fibrosis. Measurement will happen over the course of 1 month.

1 month
PsA burden in sputum at various timepoints
6 months
Safety and tolerability

Trial Safety

Trial Design

2 Treatment Groups

BX004-A
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 32 total participants across 2 different treatment groups

This trial involves 2 different treatments. BX004-A is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

BX004-A
Drug
Participants will be randomized to receive standard dose of nebulized bacteriophage
Placebo
Drug
Participants will be randomized to receive nebulized placebo

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 6 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 6 months for reporting.

Closest Location

Central Florida Pulmonary Group - Orlando, FL

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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

Patient Q&A Section

What is pseudomonas infections?

"P aeruginosa infections can cause serious infections in patients who are critically ill such as in acute or chronic obstructive pulmonary diseases, cystic fibrosis, burn victims, patients after renal [transplant](https://www.withpower.com/clinical-trials/transplant)ation or following lung transplantation. The overall mortality rate attributable to P aeruginosa infections is 12-40%. Patients with chronic kidney disease are at higher risk of developing severe P aeruginosa infection and sepsis. P aeruginosa infections frequently occur as secondary infections following hospital-acquired infections in intensive care units, in patients following surgeries and dialysis. Infection in these patients often proves fatal." - Anonymous Online Contributor

Unverified Answer

What are common treatments for pseudomonas infections?

"Infection with Pseudomonas aeruginosa is a common cause of wound infection. The antibiotic of choice is usually an aminoglycoside, either gentamicin or tobramycin. Treating all pseudomonal wound infections that are not responding to an adequate antibiotic regimen should prompt a Gram-negatives culture from a wound site." - Anonymous Online Contributor

Unverified Answer

Can pseudomonas infections be cured?

"Currently, there is no treatment for Pseudomonas infections. As it is difficult and expensive to test potential treatment options on a large scale and often costly, there is no guarantee of the best remedy to be employed for the patient, as well as inadequate understanding of the underlying mechanisms of disease. Hence, there is no guarantee that patients who receive ineffective treatment will recover any residual immunity.\n" - Anonymous Online Contributor

Unverified Answer

What causes pseudomonas infections?

"Pseudomonas may enter the body in order to defend against invading bacteria (especially those that enter by inhalation). In many cases, pseudomonas bacteria multiply and spread within the body. The most common and frequent sites of infection are the lungs and bronchial tubes. A diagnosis of pseudo-progressive respiratory distress syndrome (i.e., pseudomonas pleurisy) may be appropriate but is not necessary for antibiotic therapy. Some patients (usually with severe lung infiltrates) may be treated with antibiotics, especially in the presence of a rapid clinical response." - Anonymous Online Contributor

Unverified Answer

How many people get pseudomonas infections a year in the United States?

"Approximately 1,100,000 people are hospitalized each year for a pseudomonas infection. In a recent study, findings may have underestimated the true scope of the problem because only hospitalized patients were examined for the presence of bacterial pathogens." - Anonymous Online Contributor

Unverified Answer

What are the signs of pseudomonas infections?

"In the early stages of illness, there are specific symptoms such as fever, chills, fatigue, weight loss, joint pain and decreased range of movement. Other symptoms may also occur. There is also a range of associated medical conditions, which can be identified on the basis of those presenting with pseudomonas infections. In more severe cases, there may be bleeding, swelling of the joints and joint effusion. Patients with pseudomonas may complain of general discomfort which can be linked to other medical conditions. The signs of pseudomonas infections include a raised white blood cell count, raised erythrocyte sedimentation rate and C-reactive protein concentration." - Anonymous Online Contributor

Unverified Answer

Has bx004-a proven to be more effective than a placebo?

"Bx004-A is safe and well tolerated and may have potential to be an efficacious treatment for severe to refractory tuberculosis. Power makes it easy to find tb clinical trials tailored to your condition, treatment, or location." - Anonymous Online Contributor

Unverified Answer

What is the latest research for pseudomonas infections?

"Pseudomonas infections, however, are a persistent infection throughout the body. P. aeruginosa is now recognized as a major cause of hospital-acquired infections. Due to increasing antibiotic resistance, the use of parenteral antibiotics (even for empiric therapy, when necessary) cannot be recommended for the treatment of pneumonia without a P. aeruginosa-specific diagnosis." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of bx004-a?

"Bx004-a is well tolerated and is efficacious against P. aeruginosa infections in animal models for lung, soft tissue, and sepsis with and without bacteremia and in those with and without the carriage of antibiotic resistance. We believe the excellent pharmacokinetics and safety profile of Bx004-a will allow this promising agent to advance to early human trials." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets pseudomonas infections?

"[In the United State] there are about [300,000 suspected cases of P. aeruginosa per year with a reported [case-fatality rate] of 0.0015%-0.015% for P. aeruginosa and about 7,000 P. aeruginosa cases with reported [case-fatality rate] ≤0.01%. In the UK general population, the mean age of developing pneumonia due to P. aeruginosa is about 60 years. In the U.S. general population, the mean age of developing pneumonia due to P. aeruginosa is about 58 years." - Anonymous Online Contributor

Unverified Answer

Is bx004-a typically used in combination with any other treatments?

"Bx004 is typically used in combination with penicillermic antibiotics (including minocycline and rifampicine) and/or tetracycline. These treatments are typically recommended when the infection has either become resistant to penicillers; when other options have become unavailable; when the patient's immunity is severely compromised; and when the infection in question is life-threatening. There also is evidence that some of these treatments (particularly penicillers and tetracycline) also will enhance the effectiveness of bx004 in eliminating Lyme. There is some controversy regarding the merits of combining any of the three treatments for Lyme infection; some physicians have expressed concern that all three may have synergistic effects." - Anonymous Online Contributor

Unverified Answer

Is bx004-a safe for people?

"All treatment participants received an IV dose of 0.25 mg/kg or 1.0 mg/kg or 2 mg/kg of Bx004-a administered two times daily with a 12- to 24-hour interval over 3 to 4 days. For people with moderate or severe renal impairment, Bx004-a was administered at 0.05 mg/kg per dose of Bx004-a. People with mild renal impairment (creatinine clearance, 30 ml/min < 30 min/min) received 1.0 mg/kg Bx004-a. Adverse drug reactions were not identified during the 42 days of treatment. There were no safety concerns after 52 weeks." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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