CLINICAL TRIALS PROFILE FOR PULMOZYME

All Clinical Trials for PULMOZYME

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00117208 ↗	Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis	Completed	Pharmaxis	Phase 2	2005-11-01	The purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.
NCT00169962 ↗	Study of Pulmozyme to Treat Severe Asthma Episodes	Completed	Genentech, Inc.	Phase 2	2004-01-01	Even with current standard ED treatments 20-25% of patients presenting to the ED with an acute asthma episode will still require hospitalization. For patients unresponsive to beta-agonists the admit rates will be higher. Of those well enough to be discharged from the ED nearly 30% will relapse within one month. More than 5,000 patients with asthma still die each year in the USA. For patients who do not respond to beta-agonists, there are relatively few treatment options for rapid improvement of symptoms and pulmonary function. Presumably, mucous secretion and plugging play an important role in the pathogenesis of severe asthma unresponsive to beta-agonists. The use of agents to promote clearance of intra-luminal secretions and mucous plugs may represent an important advance in the management of acutely ill asthmatics, both to hasten recovery and prevent deterioration in the acute care setting and to prevent relapse after discharge from the ED. OBJECTIVES 2.1 Study Hypothesis: rhDNAse can be safely used in patients presenting to the Emergency Department with acute moderate-severe asthma who do not have adequate responses to beta-agonists Project Specific Aim: This is a pilot study to determine the safety of three different doses of pulmozyme® (2.5mg, 5.0mg and 7.5mg) in patients presenting to the ED with acute asthma. In addition to safety trends for improvement in pulmonary function and clinical outcomes will be monitored and data analyzed. Based on the safety profile and observable responses to treatment, this information may be used to develop larger trials to determine the efficacy and dosing strategy for treating acutely ill asthmatics with rhDNAse.
NCT00169962 ↗	Study of Pulmozyme to Treat Severe Asthma Episodes	Completed	Northwell Health	Phase 2	2004-01-01	Even with current standard ED treatments 20-25% of patients presenting to the ED with an acute asthma episode will still require hospitalization. For patients unresponsive to beta-agonists the admit rates will be higher. Of those well enough to be discharged from the ED nearly 30% will relapse within one month. More than 5,000 patients with asthma still die each year in the USA. For patients who do not respond to beta-agonists, there are relatively few treatment options for rapid improvement of symptoms and pulmonary function. Presumably, mucous secretion and plugging play an important role in the pathogenesis of severe asthma unresponsive to beta-agonists. The use of agents to promote clearance of intra-luminal secretions and mucous plugs may represent an important advance in the management of acutely ill asthmatics, both to hasten recovery and prevent deterioration in the acute care setting and to prevent relapse after discharge from the ED. OBJECTIVES 2.1 Study Hypothesis: rhDNAse can be safely used in patients presenting to the Emergency Department with acute moderate-severe asthma who do not have adequate responses to beta-agonists Project Specific Aim: This is a pilot study to determine the safety of three different doses of pulmozyme® (2.5mg, 5.0mg and 7.5mg) in patients presenting to the ED with acute asthma. In addition to safety trends for improvement in pulmonary function and clinical outcomes will be monitored and data analyzed. Based on the safety profile and observable responses to treatment, this information may be used to develop larger trials to determine the efficacy and dosing strategy for treating acutely ill asthmatics with rhDNAse.
NCT00179998 ↗	Effectiveness of Pulmozyme in Infants With Cystic Fibrosis	Completed	Genentech, Inc.	Phase 2	2005-01-01	This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
NCT00179998 ↗	Effectiveness of Pulmozyme in Infants With Cystic Fibrosis	Completed	Nationwide Children's Hospital	Phase 2	2005-01-01	This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
NCT00265434 ↗	Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis	Completed	Dr. Gerlind Schneider	Phase 3	2005-12-01	Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of Chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections. The prospective placebo controlled cross-over study aims at the evaluation of the efficacy of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.
NCT00265434 ↗	Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis	Completed	PD Dr. Assen Koitschev, Tübingen	Phase 3	2005-12-01	Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of Chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections. The prospective placebo controlled cross-over study aims at the evaluation of the efficacy of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.

Clinical Trial Conditions for PULMOZYME

Condition Name

Condition Name for PULMOZYME
Intervention	Trials
Cystic Fibrosis	12
Ischemic Stroke	3
Dry Eye	2
Sinusitis	2

Condition MeSH

Condition MeSH for PULMOZYME
Intervention	Trials
Cystic Fibrosis	13
Fibrosis	13
COVID-19	5
Infections	4

Clinical Trial Locations for PULMOZYME

Trials by Country

Trials by Country for PULMOZYME
Location	Trials
United States	69
Germany	4
Canada	2
Australia	2
Belgium	1

Trials by US State

Trials by US State for PULMOZYME
Location	Trials
Illinois	5
New York	5
North Carolina	4
Colorado	4
Texas	3

Clinical Trial Progress for PULMOZYME

Clinical Trial Phase

Clinical Trial Phase for PULMOZYME
Clinical Trial Phase	Trials
Phase 4	8
Phase 3	4
Phase 2	12

Clinical Trial Status

Clinical Trial Status for PULMOZYME
Clinical Trial Phase	Trials
Completed	14
Terminated	5
Not yet recruiting	5

Clinical Trial Sponsors for PULMOZYME

Sponsor Name

Sponsor Name for PULMOZYME
Sponsor	Trials
Genentech, Inc.	12
Northwestern University	2
McGill University Health Centre/Research Institute of the McGill University Health Centre	2

Sponsor Type

Sponsor Type for PULMOZYME
Sponsor	Trials
Other	67
Industry	16
NIH	1

PULMOZYME Market Analysis and Financial Projection

PULMOZYME: Clinical Trials, Market Analysis, and Projections

Introduction to PULMOZYME

PULMOZYME, also known as dornase alfa, is a recombinant form of human deoxyribonuclease I (rhDNase) produced by genetically engineered Chinese hamster ovary cells. It is specifically designed to manage cystic fibrosis (CF) by reducing the viscoelasticity of sputum, thereby improving pulmonary function and decreasing the risk of respiratory infections[2][5].

Mechanism of Action

PULMOZYME works by hydrolyzing the DNA in the sputum of CF patients, which is rich in extracellular DNA from degenerating leukocytes. This action reduces the viscoelasticity of the sputum, facilitating its clearance from the airways and improving lung function. This mechanism is crucial in managing the chronic respiratory infections and pulmonary complications associated with cystic fibrosis[2][5].

Clinical Trials

Efficacy and Safety in Cystic Fibrosis

A prospective, randomized, placebo-controlled study evaluated the effects of once-daily and twice-daily administration of PULMOZYME on CF patients. The study involved 968 patients aged 5 years and older with a forced vital capacity (FVC) of at least 40% of predicted. The results showed a reduction in respiratory infections requiring parenteral antibiotics and an improvement in lung function, as measured by forced expiratory volume (FEV1) and FVC[1].

Pediatric Patients

The safety and efficacy of PULMOZYME in pediatric patients were studied in a trial involving 98 patients aged 3 months to 10 years. The trial used the PARI BABY reusable nebulizer for younger patients unable to use a mouthpiece. The adverse reaction profile was similar to that in older patients, with higher incidence of cough and rhinitis in the younger age group[1].

Biosimilar Comparison

A Phase III clinical trial compared the efficacy and safety of PULMOZYME with its biosimilar, Tigerase, in CF patients. The study found that both treatments had comparable outcomes in terms of FEV1, FVC, and safety parameters, supporting the biosimilarity of Tigerase to PULMOZYME[3].

COVID-19 Pneumonia

In a proof-of-concept study, nebulized dornase alfa (PULMOZYME) was evaluated for its anti-inflammatory effects in hospitalized patients with COVID-19 pneumonia. The study showed that when combined with dexamethasone, nebulized dornase alfa reduced circulating C-reactive protein (CRP) levels, indicating its potential as an anti-inflammatory treatment in this context[4].

Market Analysis

Current Market Scenario

PULMOZYME is a well-established treatment for cystic fibrosis, with a strong presence in the major markets including the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The market forecast indicates continued growth driven by increasing healthcare spending and extensive research in cystic fibrosis treatments[2].

Market Forecast

The market forecast for PULMOZYME from 2019 to 2032 suggests significant growth, driven by the expanding patient population and the lack of alternative treatments that match its efficacy. The report provides detailed market size projections for each major market, highlighting the potential for PULMOZYME to maintain its market dominance despite the emergence of new therapies[2].

Competitive Landscape

The competitive landscape for cystic fibrosis treatments is evolving, with several emerging therapies expected to challenge PULMOZYME's market position. However, PULMOZYME's established track record and the lack of significant side effects make it a preferred choice for many patients. The report includes a comprehensive SWOT analysis and analyst views, providing insights into the challenges and opportunities that could influence PULMOZYME's market dominance[2].

Regulatory Milestones and Development Activities

PULMOZYME has undergone extensive regulatory scrutiny and has been approved for use in conjunction with standard therapies for cystic fibrosis. The drug has been studied in various clinical trials, and its safety and efficacy have been well-documented. Regulatory milestones include approvals in multiple countries and ongoing monitoring for safety and effectiveness[1][2].

Safety and Adverse Reactions

PULMOZYME is generally well-tolerated, but common adverse reactions include voice alteration, pharyngitis, rash, laryngitis, chest pain, conjunctivitis, rhinitis, and dyspnea. There have been no reports of anaphylaxis attributed to PULMOZYME. Mild to moderate urticaria and skin rash have been observed but are transient[1].

Market Projections and Future Outlook

The future market for PULMOZYME looks promising, with projected sales growth driven by increasing awareness and diagnosis of cystic fibrosis, as well as the drug's established efficacy. However, the emergence of new therapies and biosimilars could pose a challenge. The report provides detailed forecasted sales data from 2023 to 2032, which will be crucial for decision-making regarding therapeutic portfolios[2].

Key Takeaways

• Mechanism of Action: PULMOZYME hydrolyzes DNA in sputum, reducing its viscoelasticity and improving lung function in CF patients.
• Clinical Trials: PULMOZYME has shown efficacy and safety in reducing respiratory infections and improving lung function in CF patients, including pediatric populations.
• Market Analysis: PULMOZYME maintains a strong market position with projected growth, despite emerging competitors.
• Regulatory Milestones: Extensive regulatory approvals and ongoing safety monitoring support its use.
• Safety Profile: Generally well-tolerated with common adverse reactions that are mostly mild.

FAQs

What is PULMOZYME used for?

PULMOZYME is used for the management of cystic fibrosis to improve pulmonary function and reduce the risk of respiratory tract infections.

How does PULMOZYME work?

PULMOZYME works by hydrolyzing the DNA in the sputum of CF patients, reducing its viscoelasticity and facilitating its clearance from the airways.

What are the common side effects of PULMOZYME?

Common side effects include voice alteration, pharyngitis, rash, laryngitis, chest pain, conjunctivitis, rhinitis, and dyspnea.

Has PULMOZYME been compared to biosimilars?

Yes, PULMOZYME has been compared to its biosimilar, Tigerase, in a Phase III clinical trial, showing comparable efficacy and safety outcomes.

What is the market outlook for PULMOZYME?

The market outlook for PULMOZYME is positive, with projected growth driven by increasing healthcare spending and the drug's established efficacy, despite the emergence of new therapies.

Sources

1. Pulmozyme Clinical Trial Information: "Clinical Trial Information for Pulmozyme® (dornase alfa) in Cystic Fibrosis" - Genentech.
2. PULMOZYME Drug Insight and Market Forecast: "PULMOZYME Drug Insight and Market Forecast - 2032" - ResearchAndMarkets.
3. Comparison of Biosimilar Tigerase and Pulmozyme: "Comparison of biosimilar Tigerase and Pulmozyme in long-term symptomatic therapy of patients with cystic fibrosis and severe pulmonary impairment" - PLOS ONE.
4. Nebulised Dornase Alfa in COVID-19 Pneumonia: "Nebulised dornase alfa reduces inflammation and improves clinical outcomes in hospitalised patients with COVID-19 pneumonia" - eLife Sciences.
5. Genentech's Pulmozyme Drug Overview: "Genentech's Pulmozyme Drug Overview & Outlook 2016-2025" - Business Wire.