Volara System for Cystic Fibrosis

Recruiting at 1 trial location

Overseen By Daniel Weiner, MD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Daniel J. Weiner

Must be taking: Hypertonic saline

Disqualifiers: Under 18, Sputum culture results

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

20 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, it requires that you are currently using hypertonic saline treatment as part of your routine therapy.

How is the Volara System treatment different from other cystic fibrosis treatments?

The Volara System is unique because it combines airway clearance therapy with nebulization, which may enhance the delivery and effectiveness of inhaled medications by improving mucociliary clearance (the process of moving mucus out of the lungs). This dual-action approach is different from standard treatments that typically focus on either airway clearance or medication delivery separately. ^{1 2 3 4 5}

What is the purpose of this trial?

This is a small pilot study with the goal of identifying a superior sputum collection method in Cystic Fibrosis patients unable to produce a sputum. Participants will use the Volara System during clinic visit in an attempt to produce sputum.

Research Team

Daniel Weiner, MD

Principal Investigator

UPMC Children's Hospital of Pittsburgh

Eligibility Criteria

This trial is for adults with Cystic Fibrosis who struggle to produce sputum. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be excluded based on factors that could interfere with the study or their safety.

Inclusion Criteria

No sputum culture results in last one year or those who were unable to produce sputum from VPEP method used in QI project 3771

I use hypertonic saline for my lung therapy.

Exclusion Criteria

I am under 18 years old.

Sputum culture results in last one year

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants use the Volara System during a clinic visit to attempt sputum production

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

Treatment Details

Interventions

• Volara MetaNeb System

Trial Overview The trial is testing the effectiveness of the Volara MetaNeb System in helping patients with Cystic Fibrosis produce sputum during a clinic visit. It's a small pilot study aimed at improving sputum collection methods.

Participant Groups

Group I: Intrapulmonary Percussive Ventilation for Non-sputum Producer Experimental Treatment 1 Intervention

Unable to produce adequate amount of lower airway bacterial sampling for culture results in last year.

Volara MetaNeb System is already approved in United States for the following indications:

Approved in United States as Volara System for:

• Mobilization of secretions
• Lung expansion therapy
• Pulmonary atelectasis treatment

Who Is Running the Clinical Trial?

Daniel J. Weiner

Lead Sponsor

Trials

1

Recruited

20+

Findings from Research

Nebulised hypertonic saline significantly improves mucociliary clearance in cystic fibrosis patients, with immediate effects observed in trials involving 143 subjects, leading to a notable increase in lung function (FEV1) by 15% compared to isotonic saline.

Despite the positive effects on mucociliary clearance and lung function, the studies were limited to a maximum duration of three weeks, and further research is needed to assess long-term benefits and safety, particularly in patients with severe lung disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nebulised hypertonic saline for cystic fibrosis. Wark, PA., McDonald, V.[2018]

The review identified only one relevant study involving 28 participants, which compared nasal irrigation alone to nasal irrigation with endoscopic sinus surgery, but the evidence was rated as very low certainty, making it unclear if surgery improves nasal symptoms or reduces complications in cystic fibrosis patients with chronic rhinosinusitis and nasal polyposis.

There was one reported episode of bleeding during surgery, which was managed without further issues, indicating that while surgery may carry risks, the immediate safety profile appears manageable.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Surgery for nasal polyposis in cystic fibrosis. Almeida, PRL., Person, OC., Puga, ME., et al.[2023]

Endonasal fenetration is recommended for patients with cystic fibrosis to enhance the efficiency of aerosol treatments on the nasal mucosa.

This method also improves the effectiveness of swab and rinsing treatments for chronic sinusitis maxillaris associated with cystic fibrosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Aerosoltherapy and operative aspects in cystic fibrotic children with sinusitis maxillaris (author's transl)]. Werner, E.[2006]

Cystic fibrosis is a genetic disease that leads to chronic lung infections and digestive issues, primarily caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, affecting 1 in 20 Caucasians.

Recent advancements in treatment, including antibiotics, enzyme replacements, and lung transplants, have significantly improved life expectancy for cystic fibrosis patients, projected to rise from 28 years in the 1990s to 40 years in the next two decades.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Mucoviscidosis: Background, risk factors, diagnosis strategy, treatment principles]. Trofor, A.[2011]

Inhaled medications like hypertonic saline and dornase alfa can significantly improve lung function in children with cystic fibrosis by enhancing mucociliary clearance and reducing pulmonary disease exacerbations, with minimal side effects.

These treatments work by rehydrating airway surfaces and breaking down extracellular DNA, which helps address airway obstruction, inflammation, and infection—key factors in the progression of lung disease in cystic fibrosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Inhaled medications in cystic fibrosis beyond antibiotics. Sepe, A., Villella, VR., Cimbalo, C., et al.[2019]

References

1. England pubmed.ncbi.nlm.nih.gov

Nebulised hypertonic saline for cystic fibrosis. [2018]

2. England pubmed.ncbi.nlm.nih.gov

Surgery for nasal polyposis in cystic fibrosis. [2023]

3. Germany pubmed.ncbi.nlm.nih.gov

[Aerosoltherapy and operative aspects in cystic fibrotic children with sinusitis maxillaris (author's transl)]. [2006]

4. Romania pubmed.ncbi.nlm.nih.gov

[Mucoviscidosis: Background, risk factors, diagnosis strategy, treatment principles]. [2011]

5. Italy pubmed.ncbi.nlm.nih.gov

Inhaled medications in cystic fibrosis beyond antibiotics. [2019]