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

NCT04468100 | Completed Phase 3

• 2 other identifiers: DRN-CFR-III348 eff date 23.06.2017
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 14

Brief 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

Conditions

Cystic Fibrosis; Fibrosis

Keywords

Dornase; Cystic Fibrosis; Fibrosis; Lung Diseases; Pancreatic Diseases; Digestive System Diseases; Respiratory Tract Diseases; Pathologic Processes; Genetic Diseases, Inborn; Tigerase®; DNase

Outcome Measures

Primary Outcomes (1)

• Change in forced expiratory volume in the first second of a forced expiratory maneuver (FEV1) compared to baseline

  Week 24 ± 1

  FEV1 - forced expiratory volume in the first second of a forced expiratory maneuver by standard spirometry

Secondary Outcomes (5)

• Change in forced lung capacity (FVC) compared to baseline

  Week 24 ± 1

• The number of exacerbations of chronic pulmonary disease

  Week 24 ± 1

• The number of days before the chronic pulmonary disease exacerbation development

  Week 24 ± 1

• Change in body weight compared to baseline

  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

  Week 24 ± 1

Study Arms (2)

Tigerase®

EXPERIMENTAL

Dornase alfa

Biological: Tigerase®

Pulmozyme®

ACTIVE COMPARATOR

Dornase alfa

Biological: Pulmozyme®

Interventions

Tigerase® BIOLOGICAL

2.5 mL (2.5 mg) dornase alfa nebulized once daily for 168 (+/-7) days

Also known as: dornase alfa

Tigerase®

Pulmozyme® BIOLOGICAL

2.5 mL (2.5 mg) dornase alfa nebulized once daily for 168 (+/-7) days

Also known as: dornase alfa

Pulmozyme®

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed Patient Informed Consent Form for participation in the study;
• Men and women 18 years and older;
• Diagnosis of cystic fibrosis, defined as the presence of disease symptoms and a positive sweat test and / or detection of 2 mutations of the MVTR gene during genotyping;
• FEV1 ≥40% and ≤100% of the proper value;
• The ability to understand the protocol requirements, to give written consent to participate in the study (including the use and transfer of information about the patient's health status related to the study).

You may not qualify if:

• Hypersensitivity to any of used study drug, to their components, as well as a history of significant allergic reactions;
• Acute respiratory infection or exacerbation of chronic pulmonary disease within 4 weeks prior to screening and without corticosteroid or antibiotic therapy;
• Concomitant diseases and conditions with potential impact the patients safety, including:
• Severe renal impairment (serum creatinine more than 1.5 times higher than the upper limit of normal);
• Severe liver impairment (alanine aminotransferase (ALT) or aspartate aminotransferase (AST) blood serum activity is 2.5 times or more higher than the upper limit of the laboratory norm);
• A history of lung transplantation or planned transplantation during this study;
• The presence of antibodies to HIV, active viral hepatitis B and / or C and / or cirrhosis in the history or detected on Screening;
• Pregnancy and lactation;
• Refusal of patients with preserved reproductive potential to use adequate contraception throughout the study and within 30 days after the end of the study;
• Patients who underwent a blood or blood components transfusion within 10 days prior to screening;
• Drug or alcohol abuse at the time of Screening or in the past;
• Patient's participation in any other clinical trials and / or administration of an experimental drug within 30 days prior to the Screening Visit.

Sponsors & Collaborators

• AO GENERIUM: lead

Study Sites (14)

Regional Clinical Hospital

Barnaul, Altayskiy Kray, 656024, Russia

Location

Republican Clinical hospital named after G.G. Kuvatov

Ufa, Bashkortostan Republic, 450005, Russia

Location

Crimean federal university named after V.I.Vernadsky

Simferopol, Crimea Republic, 295007, Russia

Location

Sverdlovsk Regional clinical hospital No. 1

Yekaterinburg, Sverdlovsk Oblast, 620102, Russia

Location

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

Kazan', Tatarstan Republic, 420012, Russia

Location

Chelyabinsk Regional Clinical hospital

Chelyabinsk, 454076, Russia

Location

City Clinical Hospital named after D.D. Pletnev

Moscow, 105077, Russia

Location

City Multidisciplinary Hospital No. 2

Saint Petersburg, 194354, Russia

Location

Regional clinical Hospital

Saratov, 410053, Russia

Location

Scientific medical center of General Medicine and pharmacologies

Stavropol, 355000, Russia

Location

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

Tomsk, 634050, Russia

Location

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

Volgograd, 400066, Russia

Location

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

Yaroslavl, 150003, Russia

Location

Clinical hospital No. 2

Yaroslavl, 150010, Russia

Location

Related Publications (1)

• Amelina EL, Krasovsky SA, Akhtyamova-Givirovskaya NE, Kashirskaya NY, Abdulganieva DI, Asherova IK, Zilber IE, Kozyreva LS, Kudelya LM, Ponomareva ND, Revel-Muroz NP, Reutskaya EM, Stepanenko TA, Seitova GN, Ukhanova OP, Magnitskaya OV, Kudlay DA, Markova OA, Gapchenko EV. Comparison of biosimilar Tigerase and Pulmozyme in long-term symptomatic therapy of patients with cystic fibrosis and severe pulmonary impairment (subgroup analysis of a Phase III randomized open-label clinical trial (NCT04468100)). PLoS One. 2021 Dec 23;16(12):e0261410. doi: 10.1371/journal.pone.0261410. eCollection 2021.

  PMID: 34941914 DERIVED

Related Links

• Efficacy and safety of the biosimilar medicinal product Tigerase® (dornase alfa) in long-term symptomatic treatment of patients with cystic fibrosis: results of a phase III clinical trial

MeSH Terms

Conditions

Cystic Fibrosis; Fibrosis; Lung Diseases; Pancreatic Diseases; Digestive System Diseases; Respiratory Tract Diseases; Pathologic Processes; Genetic Diseases, Inborn

Interventions

dornase alfa

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Pathological Conditions, Signs and Symptoms

Study Officials

• Oksana A. Markova, MD

  AO GENERIUM

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2020
• First Posted: July 13, 2020
• Study Start: August 30, 2017
• Primary Completion: May 16, 2018
• Study Completion: May 16, 2018
• Last Updated: July 13, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

RU (14)