Efficacy and Safety of Romiplostim N01 Combined With Rituximab in Patients With Oral TPO-RA-Refractory Primary Immune Thrombocytopenia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Romiplostim N01, Rituximab.
- Who it may be relevant to
- Registry conditions: Primacy Immune Thrombocytopenia. Basic parameters: from 14 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
An Open-label, Single-arm, Single-center Study to Evaluate the Efficacy and Safety of Romiplostim N01 in Combination With Rituximab in Patients With Primary Immune Thrombocytopenia Refractory to Oral TPO-RAs
Overview
The primary objective of this trial is to assess the efficacy and safety of combining Romiplostim N01 with Rituximab for the treatment of adult patients with primary immune thrombocytopenia (ITP) whose disease is refractory to oral TPO-RAs. All participants in this study will receive the same combination treatment: Rituximab: Given once a week through an intravenous infusion for 4 weeks. Romiplostim N01: Given as a weekly injection，the dose may be adjusted each week based on the patient's platelet count. Participants will be asked to: Visit the clinic regularly for check-ups, blood tests (to monitor platelet counts), and safety assessments. Report their bleeding symptoms for evaluation. This is an open-label, single-arm trial, meaning that all participants will receive the investigational treatment, and both the research team and participants will be aware of the treatment assigned. The study aims to enroll approximately 30 adult patients aged 14 years or older who have been diagnosed with ITP and have not responded adequately to prior oral TPO-RAs (eltrombopag/hetrombopag).
Interventions
- Drug Romiplostim N01
Romiplostim N01 will be administered via subcutaneous injection once weekly based on the platele count. The starting dose is set at 3 μg/kg. Weekly dose adjustments will be guided by platelet counts measured immediately prior to administration, as follows: For a platelet count below 50 × 10⁹/L, the dose will be increased by 1 μg/kg. For a platelet count between 50 and 200 × 10⁹/L, the current dose will be maintained as the minimum therapeutic dose for bleeding risk reduction. For a platelet c - Drug Rituximab
Rituximab will be administered in accordance with the Chinese guidelines for the diagnosis and management of adult primary immune thrombocytopenia (2020 edition). The dosage will be 375 mg/m² per infusion, administered via intravenous infusion once weekly for a total of four weeks.
Primary outcome measures
- Efficacy evaluation [Time frame: from week 2 to month 12]
- Incidence of severe Adverse Events [Time frame: up to 12 months]
Secondary outcome measures (4)
- Long-term Sustained Response Rate versus Relapse Rate [Time frame: at 6 months and 12 months]
- Proportion of Participants Achieving a Rapid Platelet Response [Time frame: within the first 8 weeks of treatment]
- Change in Bleeding Score Over Time [Time frame: up to 12 months]
- Incidence of other adverse events [Time frame: up to 12 months]
Eligibility criteria
Inclusion criteria
Confirmed diagnosis of primary immune thrombocytopenia (ITP).
Age ≥14 years.
Prior exposure to at least one ITP-directed therapy (e.g., corticosteroids, intravenous immunoglobulin, recombinant human thrombopoietin, TPO-RAs, immunosuppressants, or splenectomy), including a minimum 4-week course of an oral TPO-RA (hetrombopag or eltrombopag) that was discontinued due to insufficient response.
Platelet count < 30 × 10⁹/L at screening, OR platelet count < 50 × 10⁹/L with concurrent clinically significant bleeding.
Stable glucocorticoid dose (e.g., prednisone or methylprednisolone, not exceeding 4 tablets daily) for at least 2 weeks, and stable dosing of any other immunosuppressants for at least 4 weeks prior to enrollment.
No receipt of intravenous immunoglobulin within 2 weeks before the first dose of study treatment.
No platelet transfusion within 1 week before the first dose of study treatment.
Exclusion criteria
Secondary ITP due to underlying conditions such as other autoimmune disorders, viral infections, or drug exposure.
Presence of active malignancy, pregnancy, significant cardiovascular or cerebrovascular disease, or history of arterial/venous thrombosis.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- The First Affiliated Hospital of Soochow University — Suzhou
Identifiers
NCT: NCT07206823 · 2025767