It is a multicenter, open-label, randomized, parallel-group study of the efficacy and safety of Tigerase® compared Pulmozyme® in patients with Cystic Fibrosis
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
100
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 168 (+/-7) days
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 168 (+/-7) days
Regional Clinical Hospital
Barnaul, Altayskiy Kray, Russia
Republican Clinical hospital named after G.G. Kuvatov
Ufa, Bashkortostan Republic, Russia
Change in forced expiratory volume in the first second of a forced expiratory maneuver (FEV1) compared to baseline
Week 24 ± 1
Time frame: FEV1 - forced expiratory volume in the first second of a forced expiratory maneuver by standard spirometry
Change in forced lung capacity (FVC) compared to baseline
FVC - forced lung capacity by standard spirometry
Time frame: Week 24 ± 1
The number of exacerbations of chronic pulmonary disease
Clinical symptoms of chronic pulmonary disease exacerbation required antibiotic therapy in CF patients, include the presence of at least 4 of 12 possible signs or symptoms 1. A change in color or amount of sputum; 2. The appearance or intensification of hemoptysis; 3. Strengthening cough; 4. Increased shortness of breath; 5. Increased malaise, fatigue or lethargy; 6. Temperature above 38 ° C; 7. Anorexia or weight loss; 8. Sinuses pain or soreness; 9. Changes in the nature of sinuses mucus; 10. Changes in chest physical examination; 11. Pulmonary function decrease at list 10% or more; 12. Radiographic changes.
Time frame: Week 24 ± 1
The number of days before the chronic pulmonary disease exacerbation development
Time frame: Week 24 ± 1
Change in body weight compared to baseline
Time frame: Week 24 ± 1
Change in the average score for the "Symptoms", "Activity", "Influence" subscales, as well as the average total score of the St. George's Respiratory Questionnaire (SGRQ), version 2.2
St. George's Respiratory Questionnaire (SGRQ), a questionnaire for patients with respiratory diseases is designed to assess the chronic obstructive pulmonary disease patients quality of life. The questionnaire consists of 76 questions, which are grouped in 3 parts: * The first part - symptoms - measures the degree of anxiety caused by respiratory symptoms. * The second part - activity - measures the limitation of mobility and physical activity. * The third part - the influence - evaluates the existing psychosocial consequences of bronchial obstruction.
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Crimean federal university named after V.I.Vernadsky
Simferopol, Crimea Republic, Russia
Sverdlovsk Regional clinical hospital No. 1
Yekaterinburg, Sverdlovsk Oblast, Russia
Kazan state medical University of Ministry of health, Hospital Therapy Department
Kazan', Tatarstan Republic, Russia
Chelyabinsk Regional Clinical hospital
Chelyabinsk, Russia
City Clinical Hospital named after D.D. Pletnev
Moscow, Russia
City Multidisciplinary Hospital No. 2
Saint Petersburg, Russia
Regional clinical Hospital
Saratov, Russia
Scientific medical center of General Medicine and pharmacologies
Stavropol, Russia
...and 4 more locations
Time frame: Week 24 ± 1