Table of Contents

• Trial overview
• Cancer-related thrombocytopenia studies
• Immune thrombocytopenia studies
• Cardiac surgery study
• Main endpoints used in the trials
• Who can participate

Trial overview

The trial data show several studies of Romiplostim in adults with low platelet counts. These studies are interventional, which means the research team gives a study treatment and measures the results. The listed studies are in Phase 2 or Phase 3, and they include completed, authorised, and ongoing/approved studies.^{[1][2][3][3][4]}

Across the trials, the main question is whether Romiplostim can help people keep platelet counts high enough to avoid treatment problems. In cancer studies, the goal is often to prevent chemotherapy delays or dose changes. In immune thrombocytopenia studies, the goal is to improve platelet response and reduce the need for more treatment.^[1][2][3][4]

Cancer-related thrombocytopenia studies

Two Phase 3 studies focus on chemotherapy-induced thrombocytopenia, which means low platelet counts caused by chemotherapy. One study enrolled adults with gastrointestinal, pancreatic, or colorectal cancer, and the other enrolled adults with non-small cell lung cancer, ovarian cancer, or breast cancer.^[1][4]

In both cancer studies, the main goal is to see whether Romiplostim helps patients receive chemotherapy on time and at full dose. The studies compare Romiplostim with placebo, which is an inactive treatment used for fair comparison. The primary outcome looks at whether chemotherapy had to be reduced, delayed, skipped, or stopped because platelet counts dropped below 100 x 10⁹/L.^[1][4]

These studies matter because chemotherapy schedules are important for cancer care. If platelet counts stay too low, treatment may need to change, and that can affect the planned cancer therapy. The trials are measuring whether Romiplostim can help avoid those changes.^[1][4]

Immune thrombocytopenia studies

Two studies focus on immune thrombocytopenia, a condition where the body attacks its own platelets. One Phase 3 study is in newly diagnosed primary immune thrombocytopenia and compares Romiplostim plus dexamethasone with dexamethasone alone.^[2]

The main outcome in that study is a sustained response off treatment, called 6mSROT-50. This means platelet counts stay at or above 50 x 10⁹/L for at least 6 months after treatment stops, without any ITP treatment or rescue treatment, and without major bleeding of WHO grade 2 or higher.^[2]

A second Phase 2 study, called T-MEM, looks at a more complex treatment plan for immune thrombocytopenia. It measures platelet counts at several visits and checks how many patients are in remission without needing therapy at later visits.^[3]

This means the research is not only asking whether platelet counts rise, but also whether people can stay well without ongoing treatment. That is important for patients who want a longer-lasting response rather than short-term control only.^[2][3]

Cardiac surgery study

One Phase 2 study looks at thrombocytopenic patients undergoing cardiac surgery. It tests Romiplostim before surgery and compares it with placebo or a sodium chloride solution used as a control treatment.^[3]

The main outcome is the lowest platelet count measured from the end of CEC to the 7th day after surgery. CEC means cardiopulmonary bypass, a machine used during some heart operations to keep blood moving while the heart is stopped.^[3]

This study is focused on how severe the platelet drop is after surgery. The trial is trying to find out whether preoperative Romiplostim can reduce the depth of thrombocytopenia after the operation.^[3]

Main endpoints used in the trials

The primary endpoint is the main result a trial measures to answer its research question. In these Romiplostim studies, the primary endpoints include platelet counts, bleeding-related outcomes, and whether chemotherapy can continue without dose changes.^{[1][2][3][3][4]}

• No thrombocytopenia-induced modification of chemotherapy means the patient can keep the planned chemotherapy schedule without dose reduction, delay, omission, or stopping treatment because of low platelets.^[1][4]

• 6mSROT-50 measures a lasting platelet response after treatment stops, showing whether the benefit continues for 6 months without other ITP treatment.^[2]

• Lowest platelet count after surgery shows how far the platelet level falls after cardiac surgery and whether the study treatment may limit that drop.^[3]

• Platelet count at follow-up visits is used in the T-MEM study to track response over time and remission without therapy.^[3]

These endpoints are practical and patient-focused. They show whether treatment helps people stay on their planned care path, avoid bleeding problems, or reach a stable response without ongoing medicine.^{[1][2][3][3][4]}

Who can participate

The listed studies include different patient groups, so eligibility depends on the condition being studied. The cancer trials include adult subjects with gastrointestinal, pancreatic, colorectal, non-small cell lung, ovarian, or breast cancer who have chemotherapy-induced thrombocytopenia.^[1][4]

The immune thrombocytopenia studies include people with newly diagnosed primary immune thrombocytopenia or immune thrombocytopenia more broadly. The cardiac surgery study includes thrombocytopenic patients who are scheduled for cardiac surgery.^[2][3][3]

Because the trials study different diseases, the entry rules are not the same in each one. A person may qualify for one study but not another, depending on age, diagnosis, and treatment plan.^{[1][2][3][3][4]}