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
Take a drug to treat lupus-related platelet problems
The goal of this clinical trial is to learn if sirolimus, added to standard background therapy (corticosteroids and hydroxychloroquine), works to treat systemic lupus erythematosus-associated immune thrombocytopenia (SLE-ITP) in adults aged 18 to 65 years. The study also aims to evaluate the long-term effectiveness and safety of sirolimus over 48 weeks. The main questions it aims to answer are: 1. Does sirolimus increase the overall response rate (complete or partial remission of thrombocytopenia) at 24 weeks compared with placebo? 2. The safety issues when taking sirolimus over 24 weeks (Phase 1) and up to 48 weeks (Phase 2). 3. How long does the treatment effect last, and what is the relapse rate during open-label extension? In Phase 1 (first 24 weeks), researchers will compare sirolimus to a placebo (an identical-looking capsule containing no active drug) to see if sirolimus works to treat SLE-ITP when both groups also receive standardized prednisone and hydroxychloroquine. In Phase 2 (weeks 24 to 48), all participants will receive open-label sirolimus to assess long-term efficacy and safety. Participants will: 1. Phase 1: Take sirolimus (1.5 mg once daily) or a placebo for 24 weeks, plus standardized prednisone (tapered according to protocol) and hydroxychloroquine (0.4 g daily) 2. Phase 2: After completing Phase 1, take open-label sirolimus (1.5 mg once daily) for an additional 24 weeks, continuing stable or tapering doses of prednisone and hydroxychloroquine 3. Visit the clinic at screening, baseline, weeks 4, 12, 24 (end of Phase 1), and then at weeks 28, 36, and 48 (end of Phase 2) for checkups, blood tests, and disease activity assessments 4. Receive telephone follow-ups at weeks 8, 16, 20, 32, 40, and 44 to report platelet counts, medication adherence, and adverse events
Low- vs High-Dose Sirolimus With Prednisolone for KHE and KMP
This randomized clinical trial evaluates if low-dose sirolimus (target trough 4-8 ng/mL) combined with prednisolone is noninferior in efficacy but superior in safety compared to standard high-dose sirolimus (target trough 10-15 ng/mL) combined with prednisolone in pediatric patients with kaposiform hemangioendothelioma and Kasabach-Merritt phenomenon (KHE with KMP), with participants randomized 1:1 to receive the assigned regimen, undergo routine blood and imaging monitoring, and be evaluated for clinical response and adverse events.
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