The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
Location status: Recruiting
Recruiting
NCT Number: NCT07206823
Efficacy and Safety of Romiplostim N01 Combined With Rituximab in Patients With Oral TPO-RA-Refractory Primary Immune Thrombocytopenia
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).
Recruiting
Interested in participating?
Request InfoKey information
Conditions
Age range
14 year and older
Sex eligibility
All sexes
Study type
Interventional
Phase
Not applicable
Primary location
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
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.
Treatment and study plan
Romiplostim N01
DrugRomiplostim 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 count between 200 and 400 × 10⁹/L, the dose will be decreased by 1 μg/kg.
For a platelet count exceeding 400 × 10⁹/L, the administration will be withheld. It is stipulated that dosing will resume at a reduction of 1 μg/kg from the previous dose once the platelet count returns to 200 × 10⁹/L or below.
Under no circumstances shall the dose exceed the maximum allowable limit of 10 μg/kg per week.
Rituximab
DrugRituximab 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 outcomes
-
Efficacy evaluation
Time frame: from week 2 to month 12
Efficacy assessments (platelet count) are performed weekly from Week 2 to Week 8, and then monthly until Month 12. Efficacy evaluation relies on time to platelet response, overall response rate, complete response rate and maintained response rate
-
Incidence of severe Adverse Events
Time frame: up to 12 months
Adverse events include the monitoring of the incidence of thrombotic events, myelofibrosis, and severe infections.
Secondary outcomes
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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
Platelet count is assessed weekly during this period
-
Change in Bleeding Score Over Time
Time frame: up to 12 months
-
Incidence of other adverse events
Time frame: up to 12 months
Recording the incidence of other adverse events, such as liver and kidney dysfunction, and hypersensitivity reactions.
Study contacts
Contact information is provided by the study sponsor or research team.
Sponsors and collaborators
Lead sponsor
The First Affiliated Hospital of Soochow University
Other
Registry information
Official study 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
Important dates
- Study start
- 2025
- Primary completion
- 2027
- Study completion
- 2027
- First posted
- Oct 3, 2025
- Registry last updated
- Dec 4, 2025
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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