In short
Rafutrombopag ethanolamine is an investigational thrombopoietin receptor agonist being studied for chemotherapy-induced thrombocytopenia (CIT), a condition where chemotherapy causes dangerously low blood platelet counts. The EU Clinical Trials Information System (CTIS) currently lists one authorised Phase 3 trial investigating this substance, with a planned enrollment of 121 participants. This trial evaluates both the pharmacokinetics and efficacy of the treatment in patients with CIT.
Key points
- Phase 3 trial authorised in the EU for chemotherapy-induced thrombocytopenia
- Planned enrollment of 121 participants in this interventional study
- Study includes a placebo-controlled comparison arm
- Primary outcomes measure platelet count recovery and ability to complete chemotherapy cycles
- Trial also evaluates pharmacokinetics in non-Asian participants
- Treatment is given orally as tablets
What is rafutrombopag ethanolamine?
Rafutrombopag ethanolamine is an investigational substance being studied for the treatment of chemotherapy-induced thrombocytopenia (CIT), a condition in which chemotherapy causes a dangerous drop in blood platelet counts, increasing the risk of bleeding. It is a thrombopoietin receptor agonist, meaning it mimics the action of thrombopoietin, a natural hormone that stimulates platelet production. The substance is currently under investigation in a Phase 3 clinical trial and does not yet have marketing authorisation based on the available registry data.
How does rafutrombopag ethanolamine work?
Rafutrombopag ethanolamine works by activating the thrombopoietin receptor on platelet-producing cells in the bone marrow. This activation stimulates the production of new platelets, which can help restore normal blood clotting function in patients whose platelet counts have been reduced by chemotherapy. The trial is also measuring the pharmacokinetics of the substance, which describes how the drug is absorbed, distributed, and eliminated from the body, including parameters such as maximum concentration (Cmax) and total drug exposure over time (AUC).
Which conditions is rafutrombopag ethanolamine studied for?
- Chemotherapy-induced thrombocytopenia (CIT) — a condition where chemotherapy causes low blood platelet counts, putting patients at risk of bleeding and often requiring dose reductions or delays in cancer treatment.
How is rafutrombopag ethanolamine given?
Rafutrombopag ethanolamine is being studied as an oral tablet. The trial record lists the intervention as "Hetrombopag Olamine (000 mg milligram(s), ORAL)", indicating that the dose is not reported in the registry. The study also includes a placebo arm using tablets without the active pharmaceutical ingredient for comparison.
What do the clinical trials show?
The EU Clinical Trials Information System lists one authorised Phase 3 interventional trial for rafutrombopag ethanolamine in chemotherapy-induced thrombocytopenia, with a planned enrollment of 121 participants. The study is divided into two parts. Part A evaluates the pharmacokinetic characteristics of hetrombopag in non-Asian participants with CIT. Part B evaluates the efficacy of hetrombopag treatment in participants with CIT.
The primary outcome measures include the proportion of treatment responders meeting three criteria: achieving a platelet count of at least 100×10⁹/L within 14 days after starting treatment, completing two consecutive chemotherapy cycles without thrombocytopenia-induced modification of any myelosuppressive agent, and not requiring any rescue therapy for thrombocytopenia during the treatment period.
What side effects are known?
No side effect data is available from the provided sources.