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Clinical Trials for Essential Thrombocythaemia

This article provides information about 5 ongoing clinical trials studying new treatments for Essential Thrombocythaemia, a rare blood disorder where the body produces too many platelets. These studies are testing medications including bomedemstat, ruxolitinib, and ropeginterferon alfa-2b across multiple European countries.

Clinical trial locations

Study Comparing Bomedemstat and Hydroxycarbamide for Patients with Essential Thrombocythemia

This study compares two treatments for patients who have not yet received medication to reduce their platelet count. The trial tests bomedemstat, also known as MK-3543, against hydroxycarbamide, which is also called hydroxyurea.

Main inclusion criteria: Participants must have a confirmed diagnosis based on World Health Organization criteria. They must not have received any prior treatment to reduce blood cell production. The bone marrow must show minimal scarring (Grade 0 or 1). Participants with controlled HIV, treated Hepatitis B with no detectable virus, or a history of Hepatitis C with no detectable virus may join.

Main exclusion criteria: Patients with medical conditions other than this blood disorder, those outside the specified age range, and individuals from vulnerable populations cannot participate.

Study focus: The trial aims to determine which treatment is more effective at managing platelet levels and reducing symptoms. Participants are randomly assigned to receive either bomedemstat or hydroxycarbamide in capsule form. The study lasts up to 36 months with regular monitoring to assess treatment response and side effects.

Investigational drug: Bomedemstat works by inhibiting an enzyme involved in blood cell production, helping to reduce platelet levels. Hydroxycarbamide interferes with DNA synthesis to slow down blood cell production.

Study on Bomedemstat for Patients with Essential Thrombocythemia Not Responding to or Intolerant of Hydroxyurea, Comparing with a Drug Combination

This trial studies bomedemstat for patients who have not responded well to hydroxyurea or cannot tolerate it. The study compares bomedemstat to other available treatments including ruxolitinib, peginterferon alfa-2a, anagrelide, or busulfan.

Main inclusion criteria: Participants must have a confirmed diagnosis with minimal bone marrow scarring. They must have a history of inadequate response to or intolerance of hydroxyurea. Platelet counts must be greater than 450,000 per microliter. Participants may have received up to 3 previous treatments including hydroxyurea.

Main exclusion criteria: Patients with different conditions, those outside the specified age range, and individuals from certain vulnerable populations cannot participate.

Study focus: The trial evaluates whether bomedemstat provides a more durable response compared to other available therapies. Participants receive either bomedemstat or one of several alternative medications, with monitoring for up to 36 months.

Investigational drug: Bomedemstat is an enzyme inhibitor targeting lysine-specific demethylase 1, which regulates blood cell production.

Study on the Safety of Bomedemstat for Patients with Myeloproliferative Neoplasms Who Participated in a Previous Bomedemstat Study

This extension study evaluates the long-term safety of bomedemstat in patients with myeloproliferative neoplasms, including this condition, who have already been taking the medication for at least six months in a previous trial.

Main inclusion criteria: Participants must have been part of a previous bomedemstat study and have tolerated the medication well with beneficial effects. They must be able to swallow pills and follow at-home dosing instructions.

Main exclusion criteria: Patients with other serious health conditions, those who are pregnant or breastfeeding, and those unable to follow study procedures cannot participate.

Study focus: The trial monitors long-term safety and tolerability of bomedemstat, tracking any adverse events and assessing whether the treatment continues to work effectively. The study is expected to continue until February 2035.

Investigational drug: Bomedemstat is taken orally in capsule form and works by inhibiting the LSD1 enzyme involved in blood cell regulation.

Study of Ruxolitinib for Patients with High-Risk Polycythemia Vera or High-Risk Essential Thrombocythemia

This trial compares ruxolitinib to the best available therapy for patients with high-risk forms of blood disorders, including this condition and polycythemia vera.

Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis according to WHO 2008 criteria. They must be classified as high risk based on factors like age over 60, previous blood clots, or high platelet count. Patients must have adequate liver and kidney function and be able to swallow oral medication.

Main exclusion criteria: Patients with other conditions, those not considered high-risk, and those outside the specified age range cannot participate.

Study focus: The study evaluates how effectively ruxolitinib controls blood cell counts and reduces symptoms compared to standard treatments. Participants are monitored at intervals including 6, 12, and 24 months. The trial is expected to conclude by December 2027.

Investigational drug: Ruxolitinib is a JAK1 and JAK2 inhibitor that blocks enzymes involved in blood cell production, available in 5 mg and 20 mg tablets.

Study on Ropeginterferon Alfa-2b for Patients with Essential Thrombocythemia Intolerant or Refractory to Other Treatments

This trial studies ropeginterferon alfa-2b for patients who cannot tolerate or do not respond to other treatments that reduce blood cell counts.

Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis including a bone marrow biopsy. They must need treatment to reduce blood cell counts but be unable to tolerate or respond to approved medications like hydroxyurea, anagrelide, or busulfan. Patients must not have received interferon treatment before and must have adequate liver function. A break of at least 14 days from previous treatments is required.

Main exclusion criteria: Patients with different conditions, those who have received interferon before, and those outside the specified age range cannot participate.

Study focus: The trial evaluates how well ropeginterferon alfa-2b manages the condition in patients with limited treatment options. The medication is given as an injection under the skin using a pre-filled pen. Participants are monitored at months 9, 12, 18, 24, 30, and 36 for blood count response and symptom improvement. The study is expected to conclude by December 2027.

Investigational drug: Ropeginterferon alfa-2b is an interferon that modulates the immune system to reduce the production of blood cells contributing to the disease.

Summary

These 5 clinical trials for Essential Thrombocythaemia are being conducted across multiple European countries, with Germany having the most diverse trial portfolio. Italy, France, Hungary, and Spain each host three or more studies, demonstrating strong research activity in these regions.

A notable focus emerges around bomedemstat, a new enzyme inhibitor, which is being tested in three separate trials covering different patient populations: those who are treatment-naive, those who have failed hydroxyurea, and those enrolled in long-term safety studies. This suggests significant research interest in this investigational medication as a potential treatment option.

The trials address various stages of treatment: first-line therapy with bomedemstat versus hydroxycarbamide, second-line options for hydroxyurea-resistant patients, and alternatives like ropeginterferon alfa-2b for patients who cannot tolerate standard treatments. Ruxolitinib is being studied specifically for high-risk patients.

All studies include rigorous monitoring for safety and effectiveness, with follow-up periods ranging from 145 days to several years. The trials aim to provide new treatment options for patients at different stages of their disease journey, particularly those who have limited alternatives due to treatment resistance or intolerance.

Ongoing Clinical Trials on Essential thrombocythaemia

  • Study on Apixaban, Rivaroxaban, and Aspirin for Preventing Blood Clots in Patients with JAK2V617F-Positive Myeloproliferative Neoplasms

    Not yet recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on Ropeginterferon Alfa-2b for Patients with Essential Thrombocythemia Intolerant or Refractory to Other Treatments

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Czechia France Germany Greece Hungary +4

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