Efficacy and Safety of Romiplostim N01 Combined With Rituximab in Patients With Oral TPO-RA-Refractory Primary Immune Thrombocytopenia

Not Applicable

Recruiting

• Conditions: Primacy Immune Thrombocytopenia
• Interventions: Drug: Romiplostim N01; Drug: Rituximab

• Registration Number: NCT07206823
• Lead Sponsor: The First Affiliated Hospital of Soochow University

Brief Summary

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 for 12 weeks. 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).

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-09
• Study Start: 2025-09-01
• Study First Submitted: 2025-09-26
• Study First Submitted QC: 2025-09-26
• Last Update Submitted: 2025-09-26
• Study First Posted: 2025-10-03
• Last Update Posted: 2025-10-03
• Primary Completion: 2027-02-28
• Study Completion: 2027-07-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Romiplostim N01 + Rituximab Combination Therapy	Romiplostim N01	-
Romiplostim N01 + Rituximab Combination Therapy	Rituximab	-

Primary Outcome Measures

Name	Time	Method
Efficacy evaluation	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	up to 12 months	Adverse events include the monitoring of the incidence of thrombotic events, myelofibrosis, and severe infections.

Secondary Outcome Measures

Name	Time	Method
Long-term Sustained Response Rate versus Relapse Rate	at 6 months and 12 months
Proportion of Participants Achieving a Rapid Platelet Response	within the first 8 weeks of treatment	Platelet count is assessed weekly during this period
Change in Bleeding Score Over Time	up to 12 months
Incidence of other adverse events	up to 12 months	Recording the incidence of other adverse events, such as liver and kidney dysfunction, and hypersensitivity reactions.

Trial Locations

Locations (1)

The First Affiliated Hospital of Soochow University; 🇨🇳 Suzhou, Jiangsu, China