Comparative Study of Clinical Efficacy and Safety of GNR-069 and Nplate in Patients With ITP

NCT05220878 | Completed Phase 3

• 2 other identifiers: RMP-ITP-III№ 407 eff. date 29 July 2021
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 18

Brief Summary

It is a phase III multicenter randomized double-blinded comparative study of clinical efficacy and safety of GNR-069 and Nplate in patients with idiopathic thrombocytopenic purpura

Conditions

Idiopathic Thrombocytopenic Purpura

Keywords

Idiopathic thrombocytopenic purpura; ITP; Thrombocytopenia; Thrombocytopoiesis; Bleeding; Hemorrhagic syndrome; Petechial rash; Ecchymosis; Fc-peptide; Recombinant DNA technology; Thrombopoietin receptors; Platelet formation; Fc fragment of human immunoglobulin IgG 1; Low platelet count; ITP treatment; Platelets; Haemorrhage; Platelet destruction; Impaired thrombopoiesis; Megakaryocytes; Autoantibodies; Splenectomy; Cytotoxic; T cells; Thrombopoietin receptor agonists; TPO-RAs; romiplostim; Nplate

Outcome Measures

Primary Outcomes (1)

• Proportion of patients achieving sustained response to treatment

  A sustained response to treatment is defined as the number of platelets ≥ 50.0 x 109/L for at least 9 out of 12 consecutive visits during the treatment period with the study or reference drug.

  26 weeks

Secondary Outcomes (7)

• Proportion of patients who achieve stable platelet count during treatment with investigational or reference drug

  26 weeks

• Time from initiation of therapy with investigational or reference drug to reaching a stable platelet count

  26 weeks

• Number of cases of emergency therapy for severe hemorrhagic syndrome during the treatment period, starting from the second week of therapy with the investigational or reference drug

  25 weeks

• Number of clinically significant bleeding episodes during the treatment period, starting from the second week of therapy with investigational or reference drug

  26 weeks

• Change in ITP-specific bleeding assessment tool (ITP-BAT) scores at last visit from baseline at screening

  26 weeks

Study Arms (2)

GNR-069

EXPERIMENTAL

Main group (80 patients) - multiple weekly subcutaneous injections of GNR-069, doses are calculated individually.

Biological: GNR-069

Nplate

ACTIVE COMPARATOR

Control group (80 patients) - multiple weekly subcutaneous injections of Nplate, doses are calculated individually.

Biological: Nplate

Interventions

GNR-069 BIOLOGICAL

Once a week as a subcutaneous injection. The initial dose is 1 mcg/kg.

Also known as: romiplostim

GNR-069

Nplate BIOLOGICAL

Once a week as a subcutaneous injection. The initial dose is 1 mcg/kg.

Also known as: romiplostim

Nplate

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written Informed Consent Form to participate in the study;
• Men and women aged 18-75 years inclusive at the time of signing the Informed Consent Form;
• Documented diagnosis of ITP with a disease duration of more than 12 months from the moment of confirmation of the diagnosis by bone marrow aspirate or biopsy results;
• A. For patients who have not had splenectomy:
• established absence/loss of response to therapy with at least one drug of fist-line treatment for ITP (which include GCs an IVIG); OR
• the occurrence of side effects during the course of therapy with the drug of the fist-line, making it impossible to use it further;
• B. For patients who underwent splenectomy:
• loss/lack of response to splenectomy;
• Thrombocytopenia ≥30.0 x 109/L - \<50.0 x 109/L with severe hemorrhagic syndrome or thrombocytopenia \<30.0 x 109/l, regardless of the presence of hemorrhagic syndrome, according to the results of platelet count conducted in a local laboratory for 7 days before the start of therapy with investigational or reference drug;
• Patients receiving GCs, azathioprine and danazole should receive these drugs in a maintenance dose for at least 4 weeks before starting therapy with investigational or reference drug;
• Consent of study participants with preserved childbearing function to use reliable methods of contraception (a combination of at least two methods, including 1 barrier method, for example, the use of a condom and spermicide) from the moment of signing the Informed Consent Form and 3 months after the last administration of investigational or reference drug.

You may not qualify if:

• Hypersensitivity to the components of investigational or reference drug or E. coli proteins ;
• Unresolved severe hemorrhagic syndrome requiring emergency treatment at the time of initiation of study or reference drug therapy ;
• Fisher-Evans Syndrome;
• Conditions with a high risk of thromboembolic complications ;
• Myelodysplastic syndrome and/or bone marrow transplantation in anamnesis;
• Deviations of clinical and laboratory parameters according to the results of studies of blood samples taken during the screening period;
• Positive test results for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV);
• Pregnancy or breastfeeding;
• Use of drugs:
• romiplostim used less than 3 weeks before treatment with study or reference drug;
• IVIG - less than 2 weeks prior to initiation of study or reference drug therapy;
• eltrombopag - used less than 2 weeks before treatment with study or reference drug, or planned to use eltrombopag while the patient is participating in this study;
• rituximab - used less than 14 weeks before treatment with study or reference drug, or planned to use rituximab while the patient is enrolled in this study;
• cyclophosphamide, cyclosporine, vincristine, vinblastine and other drugs used to treat ITP not listed above and not included in the list of drugs approved for use during the study - use less than 8 weeks before the start of therapy with the study or reference drug or the use of any of these drugs is planned during the patient's participation in this study;
• preparations of any hematopoietic growth factors - use less than 8 weeks before the start of therapy with an investigational or reference drug;

Sponsors & Collaborators

• AO GENERIUM: lead

Study Sites (18)

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

Ufa, Bashkortostan Republic, 450008, Russia

Location

State Budgetary Institution of Healthcare Irkutsk awarded the "Sign of Honor" Order Regional Clinical Hospital

Irkutsk, Irkutsk Oblast, 664079, Russia

Location

State Budgetary Health Institution of the Kaluga Region "Kaluga Regional Clinical Hospital"

Kaluga, Kaluga Oblast, 248007, Russia

Location

State Autonomous Healthcare Institution "Kuzbass Clinical Hospital named after S.V. Belyaev"

Kemerovo, Kemerovo Oblast, 650066, Russia

Location

State budgetary institution of health care of the Nizhny Novgorod region "Nizhny Novgorod Regional Clinical Hospital named after N. A. Semashko"

Nizhny Novgorod, Nizhny Novgorod Oblast, 603126, Russia

Location

Llc "Medis"

Nizhny Novgorod, Nizhny Novgorod Oblast, 603137, Russia

Location

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

Novosibirsk, Novosibirsk Oblast, 630091, Russia

Location

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

Rostov-on-Don, Rostov Oblast, 344022, Russia

Location

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

Samara, Samara Oblast, 443099, Russia

Location

Federal State Budgetary Educational Institution of Higher Education "Saratov State Medical University named after V.I. Razumovsky" of the Ministry of Health of the Russian Federation

Saratov, Saratov Oblast, 410012, Russia

Location

State Health Institution of the Tula Region "Tula Regional Clinical Hospital"

Tula, Tula Oblast, 300053, Russia

Location

State Budgetary Institution of Healthcare of the city of Moscow "Moscow Clinical Scientific and Practical Center named after A.S. Loginov" Department of Health of the city of Moscow.

Moscow, 111123, Russia

Location

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

Moscow, 125167, Russia

Location

State budgetary health care institution of the city of Moscow "Clinical Hospital named after S.P. Botkin" of the Department of Health of the city of Moscow

Moscow, 125284, Russia

Location

State budgetary health care institution of the city of Moscow "City Clinical Hospital named after V.V. Veresaev of the Department of Health of the City of Moscow"

Moscow, 127644, Russia

Location

State Budgetary Health Institution of the Moscow Region "Moscow Regional Research Clinical Institute named after M.F. Vladimirsky"

Moscow, 129110, Russia

Location

Federal State Budgetary Institution "National Medical Research Center named after V.A. Almazov" of the Ministry of Health of the Russian Federation

Saint Petersburg, 197341, Russia

Location

Municipal budgetary institution "Central City Hospital No. 7"

Yekaterinburg, 620137, Russia

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Hemorrhage; Purpura; Ecchymosis

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Oksana A. Markova, MD, MSc

  AO GENERIUM

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double-blinded
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Parallel assignment

Study Record Dates

• First Submitted: January 21, 2022
• First Posted: February 2, 2022
• Study Start: September 9, 2021
• Primary Completion: August 22, 2023
• Study Completion: October 19, 2023
• Last Updated: March 6, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

RU (18)