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Cystic FibrosisPhase 3NCT04468100

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

A Phase 3 study testing Tigerase® and Pulmozyme® in people with Cystic Fibrosis and Fibrosis, run by AO GENERIUM at 14 sites in Russia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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 Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cystic Fibrosis, Fibrosis
What is being tested?
Tigerase® and Pulmozyme®
Could I receive a placebo?
No, everyone receives an active treatment
How many people
100 participants
Where
14 sitesRussia
Study dates
30 Aug 2017to 16 May 2018
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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).

Who cannot join

  • 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;
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  • 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.

Trial locations and contacts

14 sites in 1 country.

14 sites in 1 country

Russia14 sites

  • Regional Clinical Hospital

    Barnaul, Altayskiy Kray, 656024

  • Chelyabinsk Regional Clinical hospital

    Chelyabinsk, 454076

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

    Kazan', Tatarstan Republic, 420012

  • City Clinical Hospital named after D.D. Pletnev

    Moscow, 105077

  • City Multidisciplinary Hospital No. 2

    Saint Petersburg, 194354

  • Regional clinical Hospital

    Saratov, 410053

  • Crimean federal university named after V.I.Vernadsky

    Simferopol, Crimea Republic, 295007

  • Scientific medical center of General Medicine and pharmacologies

    Stavropol, 355000

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

    Tomsk, 634050

  • Republican Clinical hospital named after G.G. Kuvatov

    Ufa, Bashkortostan Republic, 450005

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

    Volgograd, 400066

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

    Yaroslavl, 150003

  • Clinical hospital No. 2

    Yaroslavl, 150010

  • Sverdlovsk Regional clinical hospital No. 1

    Yekaterinburg, Sverdlovsk Oblast, 620102

Other trials in Russia

About this study

Official title: 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

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

Read the 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.

Being tested
Tigerase®, Pulmozyme®
Keywords
Dornase, Cystic Fibrosis, Fibrosis, Lung Diseases, Pancreatic Diseases, Digestive System Diseases, Respiratory Tract Diseases, Pathologic Processes, Genetic Diseases, Inborn, Tigerase®, DNase
Medical subject headings
Cystic Fibrosis, Fibrosis, Lung Diseases, Pancreatic Diseases, Digestive System Diseases, Respiratory Tract Diseases, Pathologic Processes, Genetic Diseases, Inborn

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Tigerase®Biological

    Also called dornase alfa

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

  • Pulmozyme®Biological

    Also called dornase alfa

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

Study groups in NCT04468100
Study groupRoleWhat they receive
Tigerase®ExperimentalDornase alfa
Receives: Biological: Tigerase®
Pulmozyme®Active comparatorDornase alfa
Receives: Biological: Pulmozyme®

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Week 24 ± 1

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

Secondary outcomes (5)

  1. Change in forced lung capacity (FVC) compared to baseline

    FVC - forced lung capacity by standard spirometry

    Measured: Week 24 ± 1

  2. 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.

    Measured: Week 24 ± 1

  3. The number of days before the chronic pulmonary disease exacerbation development

    Measured: Week 24 ± 1

  4. Change in body weight compared to baseline

    Measured: Week 24 ± 1

  5. 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.

    Measured: Week 24 ± 1

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Jul 2020
  2. Study start30 Aug 2017
  3. Primary completion16 May 2018
  4. Study completion16 May 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
100 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04468100
Sponsor's protocol ID
DRN-CFR-III
Other IDs
348 eff date 23.06.2017
Status last verified
Jul 2020
First submitted
8 Jul 2020
Last updated
13 Jul 2020
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 34941914

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Tigerase® and Pulmozyme® in people with Cystic Fibrosis and Fibrosis, run by AO GENERIUM at 14 sites in Russia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in forced expiratory volume in the first second of a forced expiratory maneuver (FEV1) compared to baseline (time frame: FEV1 - forced expiratory volume in the first second of a forced expiratory maneuver by standard spirometry). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04468100, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jul 2020; AzTrials last refreshed its copy on 8 Nov 2026.

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