Romiplostim N01 plus rituximab for people with primary immune thrombocytopenia unresponsive to oral TPO‑RAs

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

This study will test whether weekly romiplostim N01 injections combined with a four‑week course of rituximab can raise platelet counts in people aged 14 and older whose ITP did not respond to oral TPO‑RAs.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 14 Years and up
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (other)
• Drugs / interventions: prednisone, Rituximab
• Locations: 1 site (Suzhou, Jiangsu)
• Trial ID: NCT07206823 on ClinicalTrials.gov

What this trial studies

This is an open‑label, single‑arm trial enrolling about 30 participants aged 14 years or older with primary ITP refractory to oral TPO‑RAs. All participants receive rituximab by intravenous infusion once weekly for four weeks and weekly romiplostim N01 injections with dose adjustments based on platelet counts. Participants will have regular clinic visits for blood tests, bleeding symptom reporting, and safety monitoring. The main goals are to determine whether this combination increases platelet counts and to monitor adverse effects.

Who should consider this trial

Good fit: Ideal candidates are people aged 14 or older with confirmed primary ITP who failed to respond to at least a four‑week course of an oral TPO‑RA and meet the study's platelet and medication‑stability criteria.

Not a fit: Patients with secondary ITP, recent IVIG or platelet transfusion, or those whose ITP responded to oral TPO‑RAs are unlikely to meet eligibility or gain benefit from this specific combination in this study.

Why it matters

Potential benefit: If successful, the combination could raise platelet counts and reduce bleeding for people who did not respond to oral TPO‑RAs, offering a new treatment option.

How similar studies have performed: Rituximab and romiplostim have each shown benefit in ITP, and combination approaches have been explored in small studies, but combined therapy is not yet widely proven.

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.

Where this trial is running

Suzhou, Jiangsu

• The First Affiliated Hospital of Soochow University — Suzhou, Jiangsu, China (RECRUITING)

Study contacts

• Study coordinator: Hong Tian

Conditions: Primacy Immune Thrombocytopenia