Clinical Trials for Thrombocytopenia (Low Platelet Count)

Thrombocytopenia is a condition defined by low platelet count. Platelets are tiny cell fragments that help your blood clot — when your levels are low, you’re at higher risk for excess bleeding under the skin or in your organs. Current thrombocytopenia treatments focus on replenishing your platelet supply through blood transfusions or using corticosteroids or other types of drugs to stop your immune system from breaking down platelets.1

As doctors and researchers continue to learn more about thrombocytopenia, they’re also developing new and better ways to treat it. For the U.S. Food and Drug Administration (FDA) to approve these treatments, they must first undergo clinical trials to determine they’re safe and effective. These studies recruit participants with thrombocytopenia to try new or previously approved therapies.

New Medications for Thrombocytopenia

Thrombocytopenia can be caused by several health conditions and exposure to certain medications. While corticosteroids are effective at increasing platelet counts and can bring about a remission after short-term use, long-term use can have some serious side effects, including eye problems, bone fractures, and high blood sugar.2 Doctors and researchers continue to look for new ways to increase platelet levels with fewer side effects.

New medications for primary immune thrombocytopenia and low platelet levels caused by heparin (blood thinner) treatment are currently being studied in clinical trials. These are all novel treatments that focus on blocking the immune system from attacking and breaking down platelets. Some are available as pills, while others are engineered proteins that must be injected.

Examples of medications in current clinical trials include:

  • PF-06835375 — thought to regulate immune cells to increase platelet count3,4
  • Rilzabrutinib — thought to increase platelet count by preventing antibody production and platelet destruction5,6, Rilzabrutinb had been granted a fast-track designation but is not yet fully approved by the FDA.
  • VLX-1005 — thought to increase platelet count by blocking inflammation that activates platelets7,8

Previously Approved Medications

Doctors and researchers also look for new ways to utilize previously approved medications for treating other diseases. The FDA requires clinical trials before these therapies can be repurposed to ensure they’re safe and effective in different settings. Additionally, medications that are already approved for treating thrombocytopenia in adults must undergo new trials to ensure they’re safe to use in infants and children.

Current therapies being studied for treating thrombocytopenia have already been approved for treating liver and bone marrow cancers. Some medications have also been approved for treating primary immune thrombocytopenia, but they haven’t been studied in drug-induced or cirrhosis-associated (liver damage-associated) thrombocytopenia yet.

These therapies work in several ways — some stimulate the soft, spongy tissue in your bones (bone marrow) to produce more platelets, while others stop your immune system from breaking down platelets.

Examples of therapies in current clinical trials include:

  • TheraSphere® Yttrium-90 glass microspheres — FDA-approved for treating hepatocellular carcinoma (liver cancer); currently being investigated for treating cirrhosis-associated thrombocytopenia9
  • Romiplostim (Nplate®) — FDA-approved for treating chronic immune thrombocytopenia; currently being studied for treating chemotherapy-induced thrombocytopenia11
  • Avatrombopag (Doptelet®) — FDA-approved for treating cirrhosis-associated thrombocytopenia; currently being studied for treating chemotherapy-induced thrombocytopenia in people with glioma (brain cancer)12
  • Avatrombopag (infants and children) — FDA-approved for treating thrombocytopenia in adults; currently being studied for treating immune thrombocytopenia in infants and children13

Joining a Thrombocytopenia Clinical Trial

Find Clinical Trials Near Me

Interested in clinical trials? Simply search by postal code and type of condition to see what’s going on in your area.


If you’re living with thrombocytopenia, you may be eligible to participate in a clinical trial. You can begin your journey by researching trials that you qualify for at locations near you. Your doctor may also help make recommendations.

The FDA requires clinical trial investigators to post their requirements for joining a study, including the inclusion and exclusion criteria. These rules are set to ensure the investigators can accurately determine whether a therapy is safe and effective and to prevent participants from becoming sick during the study. To join a thrombocytopenia clinical trial, you may have to meet an age requirement, have a certain platelet count, and not have any underlying heart problems.

Choosing to take part in a clinical trial is a major decision. No matter your choice, it should be in the best interest of you and your health.