Study of the Safety and Immunogenicity of Long-term GNR-069 Therapy in ITP Patients

Extension Study of Long-term Safety and Immunogenicity of GNR-069 Patients With Idiopathic Thrombocytopenic Purpura Who Completed Phase 3 RMP-ITP-III Clinical Trial

It is a phase III extension study to assess safety and immunogenicity of long-term therapy with GNR-069 in patients with idiopathic thrombocytopenic purpura

Study Overview

• Status: Completed
• Conditions: Idiopathic Thrombocytopenic Purpura
• Intervention / Treatment: Biological: GNR-069

Detailed Description

It is an extension study in patients with ITP who completed participation in RMP-ITP-III clinical trial.

The study will be conducted in three stages:

• Transition visit - 1 day (transition of clinical trial participants from the RMP-ITP-III study to the RMP-ITP-III-X study);
• Treatment period - minimum 26 weeks;
• Follow-up period - 1 week. Patients will then be provided with the study therapy till the product market access with only safety data collection.

• Study Type: Interventional
• Enrollment (Actual): 160
• Phase: Phase 3

Contacts and Locations

Study Locations

• Russian Federation
  • Ekaterinburg, Russian Federation, 620137
    • Municipal budgetary institution "Central City Hospital No. 7"
  • Moscow, Russian Federation, 111123
    • 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, Russian Federation, 125167
    • Federal State Budget Funded Institution National Medical Research Center of Hematology, Ministry of Health of the Russian Federation (MoH of Russia)
  • Moscow, Russian Federation, 127644
    • 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"
  • Saint Petersburg, Russian Federation, 191014
    • Federal State Budgetary Institution "National Medical Research Center named after V.A. Almazov" of the Ministry of Health of the Russian Federation
  • Irkutsk Region
    • Irkutsk, Irkutsk Region, Russian Federation, 664079
      • State Budgetary Institution of Healthcare Irkutsk awarded the "Sign of Honor" Order Regional Clinical Hospital
  • Kaluga Region
    • Kaluga, Kaluga Region, Russian Federation, 248007
      • State Budgetary Health Institution of the Kaluga Region "Kaluga Regional Clinical Hospital"
  • Nizhny Novgorod Region
    • Nizhny Novgorod, Nizhny Novgorod Region, Russian Federation, 603126
      • 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 Region, Russian Federation, 603137
      • LLC "Medis"
  • Novosibirsk Region
    • Novosibirsk, Novosibirsk Region, Russian Federation, 630091
      • Federal State Budgetary Educational Institution of Higher Education "Novosibirsk State Medical University" of the Ministry of Health of the Russian Federation
  • Republic Of Bashkortostan
    • Ufa, Republic Of Bashkortostan, Russian Federation, 450008
      • Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Health of the Russian Federation
  • Rostov Region
    • Rostov-on-Don, Rostov Region, Russian Federation, 344022
      • Federal State Budgetary Educational Institution of Higher Education "Rostov State Medical University" of the Ministry of Health of the Russian Federation
  • Tula Region
    • Tula, Tula Region, Russian Federation, 300053
      • State Health Institution of the Tula Region "Tula Regional Clinical Hospital"

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Completion of participation in the study RMP-ITP-III while maintaining the clinical effectiveness of romiplostim therapy

Exclusion Criteria:

• Hypersensitivity to the components of the study drug or E. Coli proteins;
• Pregnancy or breastfeeding;
• Any diseases and conditions that, in the opinion of the Investigator, may hinder the patient's participation in the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GNR-069; Weekly subcutaneous injections of GNR-069 in the individually titrated dose.	Biological: GNR-069; Once a week as a subcutaneous injection.; Other Names: romiplostim

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency and severity of adverse events associated with the use of the GNR-069	Adverse events will be assessed based on complaints, physical examination and laboratory data.	up to 29 weeks
Number of undesirable events of particular interest in the study (Bleeding;Thrombotic/thromboembolic events)	The event of particular interest in this study are bleeding and thrombotic/thromboembolic events of any location.	up to 29 weeks
Number of clinically significant bleeding on Visits 1-5	The clinically significant bleeding will be considered events ≥ grade 2 according to CTCAE 5.0.	up to 29 weeks
Number and proportion of the patient with antidrug antibodies.	The antidrug antibodies willl be characterized by their type, titer and neutralizing activity.	up to 29 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of the loss of the treatment response	Loss of the treatment response is assessed as absense of platelet increase ≥ 50,0 х 109/L on the 4 weeks treatment with maximal study drug dose.	up to 27 weeks
Platelet count dynamics at Visits 1-5	The platalets must be counted till week 5	up to 5 weeks
Сhange in ITP-BAT bleeding scores from baseline to week 27	ITP-specific bleeding assessment tool (ITP-BAT) will be calculated at baseline and at week 27.	up to 27 weeks

Collaborators and Investigators

• Sponsor: AO GENERIUM
• Investigators: Study Chair: Oksana A. Markova, MD, MSc, AO GENERIUM

Study record dates

Study Major Dates

• Study Start (Actual): March 28, 2022
• Primary Completion (Actual): April 3, 2024
• Study Completion (Actual): April 3, 2024

Study Registration Dates

• First Submitted: August 3, 2022
• First Submitted That Met QC Criteria: August 5, 2022
• First Posted (Actual): August 8, 2022

Study Record Updates

• Last Update Posted (Actual): June 21, 2024
• Last Update Submitted That Met QC Criteria: June 20, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Bleeding; Haemorrhage; Platelets; romiplostim; ITP; Autoantibodies; T cells; Thrombocytopenia; Idiopathic thrombocytopenic purpura; Thrombocytopoiesis; 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; Platelet destruction; Impaired thrombopoiesis; Megakaryocytes; Splenectomy; Cytotoxic; Thrombopoietin receptor agonists; Thrombopoietin receptor mimetic; TPO-RAs; Nplate
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Cytopenia; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: RMP-ITP-III-X

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No