Thrombocytopenia means low platelet counts, which can cause easy bruising, bleeding gums, or internal bleeding. It has many causes — from immune disorders like ITP to chemotherapy side effects to bone marrow diseases. Treatment depends on the underlying cause and ranges from watching and waiting to steroids, immunosuppressants, or drugs that stimulate platelet production.
What's actually going on in research
Trials are testing newer thrombopoietin receptor agonists that boost platelet production, spleen tyrosine kinase inhibitors like fostamatinib for immune causes, and targeted immunosuppressants. Studies are also exploring ways to reduce bleeding risk without raising platelet counts too high, and testing treatments for drug-induced and chemotherapy-related thrombocytopenia.
TPO receptor agonists
Drugs like avatrombopag and lusutrombopag stimulate the bone marrow to make more platelets. Trials are testing them in chronic liver disease, chemotherapy patients, and other causes of low platelets.
SYK inhibitors
Fostamatinib blocks an enzyme involved in destroying platelets in immune thrombocytopenia. Studies are looking at whether similar drugs can work in other autoimmune causes of low platelets.
Bleeding prevention
Some trials focus on preventing bleeding episodes rather than just raising platelet counts. These include drugs that stabilize blood vessels or improve clotting without increasing thrombosis risk.
What to know before you search
Eligibility typically depends on platelet count levels, the underlying cause of thrombocytopenia, prior treatments tried, and bleeding history.
What types of trials are currently open
- Drug trials for ITP — Testing new immunosuppressants or platelet-boosting drugs in people with immune thrombocytopenia who haven't responded to standard treatments.
- Chemotherapy-related trials — Testing drugs that prevent or treat low platelets caused by cancer treatment, aiming to avoid transfusions and treatment delays.
- Bleeding prevention studies — Testing strategies to reduce bleeding risk in people with chronic low platelet counts, including drugs that protect blood vessel walls.
- Platelet production trials — Testing drugs that stimulate the bone marrow to make more platelets in various conditions, from liver disease to bone marrow failure.
- Observational studies — Tracking people with thrombocytopenia over time to understand which treatments work best and what predicts bleeding events.
Recently added Thrombocytopenia trials
SG301-SC Injection Safety Study in Subjects With Primary ITP Patients
This is a multicenter, randomized, double-blind, placebo-controlled Phase II clinical study. The primary objective is to evaluate the safety and efficacy of SG301 SC Injection in participants with ITP, while the secondary objectives are to assess the pharmacokinetic, pharmacodynamic and immunogenicity profiles of SG301 SC Injection in ITP participants. A total of 60 ITP participants are planned to be enrolled in this study and randomly assigned at a 1:1:1 ratio to the low dose group, high dose group and placebo group, with approximately 20 participants per group. All participants enrolled in this study may receive concomitant background therapies at stable doses for at least 4 weeks prior to the first study drug administration, and is expected to be maintained throughout the study period.
Take immune-suppressing medication at different dose levels for a rare childhood condition
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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