A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)

NCT ID: NCT04679103

Last Updated: 2020-12-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-05-03

Study Completion Date

2020-04-20

Brief Summary

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A safety and immunogenicity study in long-term treatment of Eculizumab (JSC "GENERIUM", Russian Federation) in patients with paroxysmal nocturnal hemoglobinuria, who have been involved earlier in the clinical trials of Eculizumab (JSC "GENERIUM", Russian Federation).

Detailed Description

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Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare, acquired, life-threatening, progressive clonal blood disease that is developed as a result of the expansion of one or more clones of hematopoietic stem cells with a somatic PIG A gene mutation. The main pathogenic mechanism of PNH development is a dysregulation of the complement system, in which there are no complement inhibitors CD55 and CD59 that are bounded by the GPI-anchor on the surface of blood cells, or there is a deficiency of them.

Eculizumab is the first drug for the pathogenetic therapy of PNH, a recombinant humanized monoclonal antibody against the C5 component of complement. Binding with high affinity to C5, eculizumab prevents the cleavage of C5 into C5a and C5b, thereby inhibiting the formation of pro-inflammatory cytokines (via C5a) and Membrane attack complex (MAC) (via C5b).

Using of eculizumab in patients with PNH leads to a significant decrease in hemolysis, the incidence of thrombosis and increasing of the quality of life of patients. It increases life expectancy with long-term using of eculizumab.

The drug Eculizumab, developed by JSC "GENERIUM", Russia, is a biological analogue (biosimilar) of the original drug Soliris®. At the present time Eculizumab (JSC GENERIUM, Russia) have already been authorized as Elizaria® in Russia.

This study is a multicenter open-label study of the safety, immunogenicity and efficacy of Eculizumab (JSC GENERIUM, Russia) in patients with paroxysmal nocturnal hemoglobinuria who completed participation in previous clinical trials of Eculizumab (JSC GENERIUM, Russia).

Visit 1 of this study is carried out on the day of the Visit of completion of a previously conducted study, or on another day, observing the terms of administration of the drug prescribed by the instructions for medical use of Eculizumab.

At this visit, all patients who signed the informed consent form and were immunized with meningococcal vaccine will begin therapy with Eculizumab (GENERIUM JSC), which will continue until the drug is registered or, as decided by the sponsoring company, until its commercial availability. When the last patient enrolled in the study has completed 6 months of therapy, which corresponds to the completion of Visit 13, an interim analysis of the safety and immunogenicity data of the study drug will be performed.

Conditions

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Paroxysmal Nocturnal Hemoglobinuria

Keywords

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eculizumab humanized monoclonal antibodies safety paroxysmal nocturnal hemoglobinuria rare disease biosimilar complement system C5 component of complement pro-inflammatory cytokines Membrane attack complex (MAC) immunogenicity

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Interventional
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

NAP

Study Groups

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Eculisumab (JSC GENERIUM, Russia)

Eculizumab

Group Type EXPERIMENTAL

Eculizumab (JSC GENERIUM, Russia)

Intervention Type BIOLOGICAL

Eculizumab

Interventions

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Eculizumab (JSC GENERIUM, Russia)

Eculizumab

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. Completion of participation in previous studies of Eculizumab (JSC "GENERIUM") while maintaining the clinical efficacy of the therapy with eculizumab.

In case of significant deviations from the protocol during participation in previous studies, the decision on the possibility of patient participation in this study is made on an individual basis in agreement with the company Sponsor.
2. Written informed consent to participate in this study.
3. Documented vaccination against meningococcal infection (serotypes A, C, Y and W135 Neisseria meningitides) with an unexpired period of validity and consent of the patient to undergo booster vaccination against meningococcal infection (serotypes A, C, Y and W135 Neisseria meningitides) while participating in this research when the previous vaccination expires.
4. Consent to use a reliable method of contraception (a combination of at least 2 methods, including 1 barrier method, for example, using a condom and spermicide), from the moment of signing the informed consent until 10 weeks after the last administration of the study drug.

Exclusion Criteria

1. Hypersensitivity to the study drug, as well as to its components.
2. Hypersensitivity to the active ingredients or any other component of the vaccine for the prevention of meningococcal infection, or a life-threatening reaction to the previous administration of a vaccine containing similar components.
3. Comorbidities and conditions that, in the opinion of the Investigator, endanger the safety of the patient while participating in the study.
4. Pregnancy or breastfeeding.
5. Alcohol, drug or drug addiction.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AO GENERIUM

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Oksana Markova, MD

Role: STUDY_CHAIR

AO GENERIUM

Locations

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State budgetary healthcare institution "Chelyabinsk Regional Clinical Hospital"

Chelyabinsk, Chelyabinsk Oblast, Russia

Site Status

Federal State Budgetary Institution of Science "Kirov Research Institute of Hematology and Blood Transfusion of the Federal Medical and Biological Agency"

Kirov, Kirov Oblast, Russia

Site Status

Federal State Budgetary Educational Institution of Higher Education "First St. Petersburg State Medical University named after Academician I.P. Pavlov"

Saint Petersburg, Leningradskaya Oblast', Russia

Site Status

Federal State Budgetary Institution "National Medical Research Center of Hematology" of the Ministry of Health of the Russian Federation

Moscow, Moscow Oblast, Russia

Site Status

State Budgetary Institution of Healthcare of the City of Moscow City Clinical Hospital named after S.P. Botkin of the Moscow Department of Health

Moscow, Moscow Oblast, Russia

Site Status

State budgetary institution of health care of the Moscow region "Moscow Regional Research Clinical Institute named after M.F. Vladimirsky"

Moscow, Moscow Oblast, Russia

Site Status

State budgetary institution of the Rostov region "Rostov Regional Clinical Hospital"

Rostov-on-Don, Rostov Oblast, Russia

Site Status

Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Health of the Russian Federation

Samara, Samara Oblast, Russia

Site Status

State Budgetary Healthcare Institution of the Sverdlovsk Region "Sverdlovsk Regional Clinical Hospital No. 1"

Yekaterinburg, Sverdlovsk Oblast, Russia

Site Status

Countries

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Russia

Other Identifiers

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№205. eff.data 03 May 2018

Identifier Type: OTHER

Identifier Source: secondary_id

ECU-PNH-III-X

Identifier Type: -

Identifier Source: org_study_id