NCT04468100

Efficacy and Safety of Tigerase® vs. Pulmozyme® in Patients With Cystic Fibrosis

A Multicenter Prospective Randomized Comparative Study of Pharmacokinetics, Clinical Efficacy and Safety of Tigerase® (JSC GENERIUM, Russia) vs. Pulmozyme® (Hoffmann-La Roche, Switzerland) as Part of Complex Therapy in Patients With Cystic Fibrosis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (actual)
• Sponsor: AO GENERIUM · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

It is a multicenter, open-label, randomized, parallel-group study of the efficacy and safety of Tigerase® compared Pulmozyme® in patients with Cystic Fibrosis

Detailed description

Cystic fibrosis (CF) is a common hereditary disease with an autosomal recessive type of inheritance, characterized by systemic damage to the exocrine glands, mainly the bronchopulmonary and gastrointestinal systems. CF is usually characterized by a severe course and poor prognosis. The severity of the disease and the life expectancy of the patient with CF is determined primarily by the state of the bronchopulmonary system; more than 90% of patients die from lung diseases. CF Pulmonary damage develops as a result of a gene mutation - cystic fibrosis transmembrane regulator of ion conductivity Na and Cl (CFTR-cystic fibrosis transmembrane regulator). The main function of CFTR is to regulate the transport of sodium and chlorine across the cell membrane and is part of the cAMP-dependent chlorine channel. CFTR-protein dysfunction in the bronchial tree epithelial cells leads to a blockage of the chlorine ions transport and an increase in the sodium ions absorption, and impaired fluid secretion through the epithelial cells apical membrane. Dornase alpha, a human recombinant deoxyribonuclease 1 (rhDNase, rhDNase) hydrolyzes extracellular DNA (viscous polyanion) that enters the sputum from destroyed neutrophils, thereby reducing the adhesion and viscosity of sputum. In CF patients dornase alpha is used as symptomatic therapy in combination with standard therapy in patients with a forced vital capacity (FVC) index of at least 40% of the proper value.

Conditions

• Cystic Fibrosis
• Fibrosis

Interventions

Type	Name	Description
BIOLOGICAL	Tigerase®	2.5 mL (2.5 mg) dornase alfa nebulized once daily for 168 (+/-7) days
BIOLOGICAL	Pulmozyme®	2.5 mL (2.5 mg) dornase alfa nebulized once daily for 168 (+/-7) days

Timeline

• Start date: 2017-08-30
• Primary completion: 2018-05-16
• Completion: 2018-05-16
• First posted: 2020-07-13
• Last updated: 2020-07-13

Locations

14 sites across 1 country: Russia