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

Last updated: July 8, 2020
Sponsor: AO GENERIUM
Overall Status: Completed

Phase

3

Condition

Lung Disease

Cystic Fibrosis

Scar Tissue

Treatment

N/A

Clinical Study ID

NCT04468100
DRN-CFR-III
348 eff date 23.06.2017
  • Ages > 18
  • All Genders

Study 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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Signed Patient Informed Consent Form for participation in the study;

  2. Men and women 18 years and older;

  3. Diagnosis of cystic fibrosis, defined as the presence of disease symptoms and apositive sweat test and / or detection of 2 mutations of the MVTR gene duringgenotyping;

  4. FEV1 ≥40% and ≤100% of the proper value;

  5. The ability to understand the protocol requirements, to give written consent toparticipate in the study (including the use and transfer of information about thepatient's health status related to the study).

Exclusion

Exclusion Criteria:

  1. Hypersensitivity to any of used study drug, to their components, as well as a historyof significant allergic reactions;

  2. Acute respiratory infection or exacerbation of chronic pulmonary disease within 4weeks prior to screening and without corticosteroid or antibiotic therapy;

  3. Concomitant diseases and conditions with potential impact the patients safety,including:

  • Severe renal impairment (serum creatinine more than 1.5 times higher than theupper limit of normal);

  • Severe liver impairment (alanine aminotransferase (ALT) or aspartateaminotransferase (AST) blood serum activity is 2.5 times or more higher than theupper limit of the laboratory norm);

  1. A history of lung transplantation or planned transplantation during this study;

  2. The presence of antibodies to HIV, active viral hepatitis B and / or C and / orcirrhosis in the history or detected on Screening;

  3. Pregnancy and lactation;

  4. Refusal of patients with preserved reproductive potential to use adequatecontraception throughout the study and within 30 days after the end of the study;

  5. Patients who underwent a blood or blood components transfusion within 10 days prior toscreening;

  6. Drug or alcohol abuse at the time of Screening or in the past;

  7. Patient's participation in any other clinical trials and / or administration of anexperimental drug within 30 days prior to the Screening Visit.

Study Design

Total Participants: 100
Study Start date:
August 30, 2017
Estimated Completion Date:
May 16, 2018

Study 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.

Connect with a study center

  • Regional Clinical Hospital

    Barnaul, Altai Region 656024
    Russian Federation

    Site Not Available

  • Crimean federal university named after V.I.Vernadsky

    Simferopol, Crimea Republic 295007
    Russian Federation

    Site Not Available

  • Republican Clinical hospital named after G.G. Kuvatov

    Ufa, Republic Of Bashkortostan 450005
    Russian Federation

    Site Not Available

  • Kazan state medical University of Ministry of health, Hospital Therapy Department

    Kazan, Republic Of Tatarstan 420012
    Russian Federation

    Site Not Available

  • Sverdlovsk Regional clinical hospital No. 1

    Ekaterinburg, Sverdlovsk Region 620102
    Russian Federation

    Site Not Available

  • Chelyabinsk Regional Clinical hospital

    Chelyabinsk, 454076
    Russian Federation

    Site Not Available

  • City Clinical Hospital named after D.D. Pletnev

    Moscow, 105077
    Russian Federation

    Site Not Available

  • City Multidisciplinary Hospital No. 2

    Saint Petersburg, 194354
    Russian Federation

    Site Not Available

  • Regional clinical Hospital

    Saratov, 410053
    Russian Federation

    Site Not Available

  • Scientific medical center of General Medicine and pharmacologies

    Stavropol, 355000
    Russian Federation

    Site Not Available

  • Tomsk national medical research Center of the Russian Academy of Sciences, Genetic Clinic Research Institute medical genetics

    Tomsk, 634050
    Russian Federation

    Site Not Available

  • Volgograd State Medical University, Department of clinical pharmacology and intensive care

    Volgograd, 400066
    Russian Federation

    Site Not Available

  • Children's Clinical Hospital No. 1 ", Cystic Fibrosis center

    Yaroslavl, 150003
    Russian Federation

    Site Not Available

  • Clinical hospital No. 2

    Yaroslavl, 150010
    Russian Federation

    Site Not Available

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