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

• Register: ClinicalTrials.gov
• Last refreshed on: 18 March 2024
• Main ID: NCT05220878
• Date of registration: 21/01/2022
• Prospective Registration: No
• Primary sponsor: AO GENERIUM
• Public title: Comparative Study of Clinical Efficacy and Safety of GNR-069 and Nplate in Patients With ITP
• Scientific title: Multicenter Randomized Double-blind Comparative Study of Clinical Efficacy and Safety of GNR-069 (JSC "GENERIUM", Russia) and Nplate (Amgen Europe BV, The Netherlands) in Patients With Idiopathic Thrombocytopenic Purpura
• Date of first enrolment: September 9, 2021
• Target sample size: 160
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT05220878
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 3

Countries of recruitment

Russian Federation

Contacts

• Name: Oksana A. Markova, MD, MSc
• Affiliation: AO GENERIUM

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Written Informed Consent Form to participate in the study;

2. Men and women aged 18-75 years inclusive at the time of signing the Informed Consent
Form;

3. 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;

4. 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;

5. 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;

6. 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;

7. 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.

Exclusion Criteria:

1. Hypersensitivity to the components of investigational or reference drug or E. coli
proteins ;

2. Unresolved severe hemorrhagic syndrome requiring emergency treatment at the time of
initiation of study or reference drug therapy ;

3. Fisher-Evans Syndrome;

4. Conditions with a high risk of thromboembolic complications ;

5. Myelodysplastic syndrome and/or bone marrow transplantation in anamnesis;

6. Deviations of clinical and laboratory parameters according to the results of studies
of blood samples taken during the screening period;

7. Positive test results for hepatitis B, hepatitis C, or human immunodeficiency virus
(HIV);

8. Pregnancy or breastfeeding;

9. 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;

- Influenza vaccines - less than 21 days prior to start of treatment with study or
reference drug;

- vaccines to prevent novel coronavirus disease (COVID-19) - completion of the
vaccination program less than 21 days prior to the start of study or reference
drug therapy;

- other vaccines - less than 8 weeks prior to start of treatment with study or
reference drug;

10. Splenectomy within 12 weeks prior to screening;

11. Participation in any clinical trials and/or use of unregistered drugs within 4 weeks
prior to screening or 5 drug half-lives (whichever is greater);

12. Any other disease or condition that, in the opinion of the investigator, may preclude
the patient from participating in the study.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Idiopathic Thrombocytopenic Purpura

Intervention(s)

Biological: GNR-069

Biological: Nplate

Primary Outcome(s)

Proportion of patients achieving sustained response to treatment [Time Frame: 26 weeks]

Secondary Outcome(s)

Proportion of patients with no/loss of response to treatment with investigational or reference drug [Time Frame: 26 weeks]

Proportion of patients receiving approved ITP prophylactic drugs (glucocorticosteroids, azathioprine, danazol) in this study at the time of randomization [Time Frame: 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 [Time Frame: 25 weeks]

Proportion of patients who achieve stable platelet count during treatment with investigational or reference drug [Time Frame: 26 weeks]

Time from initiation of therapy with investigational or reference drug to reaching a stable platelet count [Time Frame: 26 weeks]

Change in ITP-specific bleeding assessment tool (ITP-BAT) scores at last visit from baseline at screening [Time Frame: 26 weeks]

Number of clinically significant bleeding episodes during the treatment period, starting from the second week of therapy with investigational or reference drug [Time Frame: 26 weeks]

Secondary ID(s)

RMP-ITP-III

? 407 eff. date 29 July 2021

Source(s) of Monetary Support

Please refer to primary and secondary sponsors