Thrombocytopenia is a low platelet count that can cause bleeding — from easy bruising to life-threatening hemorrhage — and has many causes including immune destruction (ITP), bone marrow problems, and drug reactions. Treatment depends entirely on the cause and severity.
What's actually going on in research
Thrombopoietin receptor agonists (romiplostim, eltrombopag) stimulate platelet production and are standard for chronic immune thrombocytopenia (ITP), and newer agents with different mechanisms are entering trials. Fostamatinib, an SYK inhibitor targeting the immune destruction of platelets, is approved and being studied in earlier lines. Gene therapy for inherited platelet disorders like Wiskott-Aldrich syndrome is in early clinical trials.
TPO receptor agonists
Drugs that stimulate bone marrow to produce more platelets — romiplostim, eltrombopag, and avatrombopag — are being tested in combination and as early treatment for ITP.
SYK inhibitors
Fostamatinib blocks the SYK kinase involved in platelet destruction by immune cells. Trials are testing it in newly diagnosed ITP and in thrombocytopenia from other causes.
Gene therapy for platelet disorders
For inherited platelet production defects like CAMT and Wiskott-Aldrich syndrome, gene correction trials are attempting to restore normal platelet numbers with a single treatment.
What to know before you search
Eligibility depends on thrombocytopenia cause (ITP, MDS, chemotherapy-induced, inherited), platelet count, bleeding symptoms, and prior treatment history.
What types of trials are currently open
- ITP drug trials — Testing thrombopoietin agonists, immunosuppressives, and SYK inhibitors for immune thrombocytopenia.
- Chemotherapy-related trials — Testing platelet growth factors and transfusion strategies to manage treatment-induced thrombocytopenia.
- Inherited disorder trials — Evaluating gene therapy for congenital thrombocytopenias.
- Rituximab and splenectomy trials — Comparing second-line options timing and sequencing in chronic ITP.
- Supportive care trials — Testing platelet transfusion thresholds and antifibrinolytic strategies.
Recently added Thrombocytopenia trials
High-Dose Dexamethasone Combined With Orelabrutinib Versus High-Dose Dexamethasone Combined With Placebo in Adult Patients With Newly Diagnosed Primary Immune Thrombocytopenia
This is a randomized controlled study of high-dose Dexamethasone combined with Orelabrutinib versus high-dose Dexamethasone combined with placebo in adult patients with newly diagnosed Primary Immune Thrombocytopenia.
Daratumumab in Immune-mediated Thrombotic Thrombocytopenic Purpura
Data about efficacy and safety of daratumumab in iTTP refractory or intolerant to standard immunosuppressive treatments are scarce. Therefore, the investigators aim at collecting evidence on a larger number of patients through a collaborative, international study.
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