Clinical Trials for Thrombocythemia (High Platelet Count)

Thrombocythemia  is a condition of increased platelet levels, which puts you at an increased risk for blood clots.1 Currently approved treatments for thrombocythemia focus on reducing the number of cells in the bone marrow to limit platelet production.

As doctors and researchers learn more about thrombocythemia, they continue to look for new medications and to find new uses for previously approved therapies. Clinical studies recruit volunteers with thrombocythemia to determine whether they’re safe and effective at reducing platelet levels to prevent blood clots.

By choosing to participate in a clinical trial, you’re taking an active part in your health and helping to advance scientific research. Some people even experience benefits from these experimental treatments. The clinical trials discussed below are just a few of current studies.

New Thrombocythemia Treatments

The current standard of care for thrombocythemia is the chemotherapy agent hydroxyurea. In more severe cases, doctors may also prescribe a low-dose Aspirin® regimen as a blood thinner to help prevent blood clots. However, some people become resistant to hydroxyurea, making it ineffective.2 Because of this, researchers have set out to find new ways of treating thrombocythemia.

IMG-7289 for Thrombocythemia

One promising therapy is IMG-7289 (bomedemstat), which has now entered phase 2 clinical trials. IMG-7289 blocks an essential enzyme used by megakaryocytes, the specialized cells in the bone marrow that produce platelets. By inhibiting megakaryocyte function, your platelet levels would drop over time.3

IMG-7289 has been granted Fast Track designation by the FDA, which expedites the development and review of drugs for treating rare conditions such as essential thrombocythemia.4 There are currently a handful of studies investigating IMG-7289 for treating essential thrombocythemia and polycythemia vera, a type of blood cancer.5-7

Peptide-Based Vaccines for Thrombocythemia

Peptide (protein)-based vaccines are gaining popularity as treatments for a variety of diseases.8 Essential thrombocythemia is commonly caused by a mutation in the CALR gene, which is responsible for controlling cell growth.9 Doctors and researchers are currently investigating a peptide-based vaccine that can boost the immune system against only the stem cells with a mutated CALR gene. Therapies like these show promise for novel ways of treating thrombocythemia without affecting all bone marrow cells.10

Previously Approved Treatments

While new therapies showcase exciting advances in medicine, doctors and researchers also look to previously approved treatments that can be used to treat other diseases. These therapies must go through clinical trials again to ensure they’re safe and effective in different settings.

Several available therapies are currently being investigated for treating thrombocythemia. Many of these are used to treat other myeloproliferative neoplasms (MPNs, semi-malignant conditions that start in the bone marrow), myelodysplastic syndromes (MDS, blood cancers caused by immature blood cells in the bone marrow), and anemia.11,12 Since these cancers and conditions are similar to one another, doctors and researchers believe these treatments may be effective in thrombocythemia.

Examples of medications currently being studied and their approved uses include:

  • Ruxolitinib (Jakavi®) — a Janus kinase (JAK) inhibitor that blocks cell growth signals in MPNs13
  • Ropeginterferon (Bestremi®)
  • Fedratinib (Inrebic®) and Pacritinib (Vonjo®) used for myelofobrosis

Joining a Thrombocythemia 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 essential thrombocythemia, you may be eligible to participate in a clinical trial. Looking to participate in one? The best place to start is by researching trials near you that you may be eligible for.

Clinical trial investigators post their requirements for joining a study online. You must meet a certain set of criteria, known as inclusion criteria, to participate. They’ll also likely have exclusion criteria, which restrict you from participating. These rules are set to keep study participants safe and ensure the trial results are accurate. To join a thrombocythemia clinical trial, you may have to:

  • Have elevated platelet levels above a certain threshold
  • Meet certain age and health and other requirements

Choosing to take part in a clinical trial is a major decision. Whatever your choice is, it should be what’s best for you.