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

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What is this study about?

This clinical trial is focused on studying a condition called Essential Thrombocythemia, which is a type of blood disorder where the body produces too many platelets. Platelets are small blood cells that help with clotting, and having too many can lead to problems like blood clots or bleeding. The study is looking at a new treatment called Bomedemstat (also known by its code name MK-3543) and comparing it to the best available therapy for patients who have not responded well to or cannot tolerate a common treatment called hydroxyurea.

The purpose of the study is to compare the effectiveness and safety of Bomedemstat against other treatments that are currently available. Participants in the study will receive either Bomedemstat or another treatment, which could include medications like Ruxolitinib, Peginterferon Alfa-2a, Anagrelide Hydrochloride Monohydrate, or Busulfan. Some participants may receive a placebo. The study will last for a period of up to 36 months, during which time participants will be monitored for their response to the treatment and any side effects they may experience.

Throughout the study, researchers will assess various outcomes, such as the rate of durable clinicohematologic response, which refers to how well the blood disorder is controlled over time. They will also look at changes in symptoms and overall health, as well as any adverse events or complications that may occur. The goal is to determine if Bomedemstat is a safe and effective option for managing Essential Thrombocythemia in patients who have limited treatment options.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including a diagnosis of essential thrombocythemia and a history of inadequate response to or intolerance of hydroxyurea.

2 initial assessment

An initial assessment is conducted to evaluate the current health status. This includes checking platelet and neutrophil counts, which must meet specific thresholds.

3 randomization

Participants are randomly assigned to receive either bomedemstat or the best available therapy. This process ensures that the study results are unbiased.

4 treatment phase

Participants receiving bomedemstat take it orally in capsule form. The dosage and frequency are determined by the study protocol.

Participants receiving the best available therapy may be given medications such as ruxolitinib, peginterferon alfa-2a, anagrelide hydrochloride monohydrate, or busulfan, depending on individual needs and previous treatments.

5 ongoing monitoring

Regular monitoring is conducted to assess the effectiveness and safety of the treatment. This includes tracking changes in symptoms and any side effects experienced.

6 evaluation of outcomes

The primary outcome measured is the durable clinicohematologic response rate. Secondary outcomes include changes in fatigue levels, symptom scores, and the occurrence of thrombotic or hemorrhagic events.

7 completion of study

The study is expected to conclude by October 2028. Participants’ health and response to treatment are evaluated to determine the overall effectiveness of bomedemstat compared to the best available therapy.

Who Can Join the Study?

  • The patient must have a diagnosis of essential thrombocythemia (ET), which is a condition where the body produces too many platelets, according to specific criteria set by the World Health Organization.
  • The patient must have a bone marrow fibrosis score of Grade 0 or Grade 1. This score is a way to measure the amount of scar tissue in the bone marrow.
  • The patient must have a history of not responding well to or not being able to tolerate hydroxyurea, a medication used to treat ET, based on specific guidelines.
  • The patient must have not responded well or lost response to their most recent ET treatment, which means they need to change their current treatment.
  • The patient must have a platelet count greater than 450,000 per microliter, which is a measure of the number of platelets in the blood, checked within 72 hours before starting the study treatment.
  • The patient must have an absolute neutrophil count (ANC) of at least 0.75 x 10^9/L, which is a measure of a type of white blood cell, checked within 72 hours before starting the study treatment.
  • The patient may have received up to 3 previous treatments, including hydroxyurea.
  • Both male and female patients can participate.
  • The study includes patients from vulnerable populations, which means those who might need extra protection or care.

Who Cannot Join the Study?

  • Patients who have a different condition than Essential Thrombocythemia with an inadequate response to or intolerance of hydroxyurea.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the specified clinical trial groups.
  • Patients who are considered part of a vulnerable population that is not eligible for this study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Universitario De Salamanca Salamanca Spain
Azienda Ospedaliero Universitaria Careggi Florence Italy
Technische Universitaet Dresden Dresden Germany
Unidade Local De Saude De Lisboa Ocidental E.P.E. Carnaxide Portugal

Other Sites

Site Name City Country Status
Pratia Hematologia Sp. z o.o. Katowice Poland
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Hospital Clinic De Barcelona Barcelona Spain
Szabolcs-Szatmar-Bereg Varmegyei Oktatokorhaz Nyiregyhaza Hungary
Region Oerebro Laen Orebro Sweden
Universitaetsklinikum Aachen AöR Aachen Germany
Centre Hospitalier Lyon Sud Pierre Benite France
Hospital Universitario 12 De Octubre Madrid Spain
Hospital Del Mar Barcelona Spain
Universita’ Degli Studi Di Ferrara Ferrara Italy
Instituto Portugues De Oncologia Do Porto Francisco Gentil E.P.E. Porto Portugal
CCAB Centro Clinico Academico Braga Associacao Braga Portugal
Hospital Universitario Dr Peset Aleixandre Valencia Spain
Albert Schweitzer Ziekenhuis Dordrecht The Netherlands
Azienda Ospedaliera Ordine Mauriziano Di Torino Turin Italy
Hospital General Universitario De Albacete Albacete Spain
Universita’ Politecnica Delle Marche Ancona Italy
Universidade De Santiago De Compostela Santiago De Compostela Spain
Azienda Socio Sanitaria Territoriale Dei Sette Laghi Varese Italy
Istituto Oncologico Veneto Padua Italy
University Of Debrecen Debrecen Hungary
Karolinska University Hospital Solna Sweden
Semmelweis University Budapest Hungary
Gasthuiszusters Antwerpen Antwerp Belgium
Hospital Costa del Sol Marbella Spain
Hospital Universitario Virgen De La Victoria Malaga Spain
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Cslpbo Hmevwhlfivs Rvbzztlv Uyaqpwlrwazoe Dd Twxgb Tours France
Cjxr Dv Ngdbq Vandoeuvre Les Nancy France
Galkyzjjttdrufcdk Vchynmhex Pdrz Aschun Eidwyttz Obbxbd Kwmdvn Gyor Hungary
Acrbbye Oikgtochwso Ngtlawtfa Sj Agkdxpp E Bhkpls E C Avpmtr Asnxxcxordu Alexandria Italy
Arbzslu Upo Icuau Dn Rpcdpp Extfjs Reggio Emilia Italy
Upeiomyncxjk Mcsrkca Copvihb Ghzotgxau Groningen The Netherlands
Szualgugn Mrwoyey Zsdsvqmcar Groningen The Netherlands
Mtzppxuowshpohfcaujovabzxs Hcpkmzvdjonmptpe Halle (Saale) Germany
Iiqfbmsf Cvzgww Dnugjhuqoaxbfwcus L'hospitalet De Llobregat Spain
Ammmrnpvzb Pergdnjl Hezfpmok Dj Pkxyp Paris France
Hgiyadty Vbob dxuqlfii Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
01.02.2024
France France
Not recruiting
01.02.2024
Germany Germany
Not recruiting
01.02.2024
Hungary Hungary
Not recruiting
01.02.2024
Italy Italy
Recruiting
01.02.2024
Poland Poland
Not recruiting
01.02.2024
Portugal Portugal
Recruiting
01.02.2024
Spain Spain
Recruiting
01.02.2024
Sweden Sweden
Not recruiting
01.02.2024
The Netherlands The Netherlands
Not recruiting
01.02.2024

Trial locations

Bomedemstat is a medication being studied for its safety and effectiveness in treating patients with essential thrombocythemia, a condition where the body produces too many platelets. This medication is being compared to other therapies to see if it can provide a better or more durable response for patients who have not responded well to or cannot tolerate another treatment called hydroxyurea.

Investigated diseases:

Essential Thrombocythemia – This is a chronic blood disorder characterized by the overproduction of platelets by the bone marrow. It often progresses slowly and may not cause symptoms initially. Over time, individuals may experience headaches, dizziness, or vision changes due to increased blood viscosity. The condition can lead to complications such as blood clots or bleeding due to the abnormal platelet function. In some cases, the disease may evolve into more severe blood disorders. Regular monitoring is essential to manage the progression and potential complications.

Trial ID:
2023-504865-21-00
Protocol code:
MK-3543-006
Trial Phase:
Therapeutic confirmatory (Phase III)

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