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A study to evaluate the effectiveness and safety of iadademstat in adults with essential thrombocythemia who do not respond well to or cannot tolerate hydroxyurea

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What is this study about?

This study is being conducted to investigate the effectiveness and safety of iadademstat in adults with essential thrombocythemia. Essential thrombocythemia is a blood disorder where the body produces too many platelets, which are the small cells in the blood responsible for clotting. This study focuses on individuals whose condition has not responded well to hydroxyurea or who cannot tolerate that medication.

The treatment being tested is iadademstat, which will be taken as an oral solution, meaning a liquid medicine swallowed by mouth. The main goal of the study is to see if this new medicine can help reduce platelet counts to a normal range and to monitor any side effects that may occur during the process.

Who Can Join the Study?

  • You must be at least 18 years old.
  • You must be able and willing to agree to all study steps, including bone marrow evaluation (checking the soft tissue inside bones where blood cells are made) and peripheral blood sampling (taking blood from a vein).
  • Women who could become pregnant must have a negative pregnancy test and agree to use contraception (methods to prevent pregnancy) during the study and for 4 months after the last dose.
  • Men must agree to use effective contraception from the start of treatment until 4 months after the last dose.
  • You must agree not to donate or freeze eggs or sperm during the study or for 4 months after the last dose.
  • You may have a history of malignancy (cancer), as long as it does not interfere with the safety or results of the study.
  • Your body weight must be at least 50 kg.
  • You must have a diagnosis of Essential Thrombocythemia (ET), which is a condition where the body makes too many platelets (cells that help blood clot).
  • You must be considered high-risk, meaning you have either a history of thrombosis (blood clots) or you have the JAK2 mutation and are older than 60.
  • You must have previously used hydroxyurea (HU), a medication used to lower blood counts, and either could not tolerate it or it did not work well enough to control your platelet count.
  • You must have stopped previous treatments for a specific amount of time: 24 hours for anagrelide or hydroxyurea, 4 weeks for interferon, and 7 days for all other treatments.
  • Your blood tests before the study must show a platelet count higher than 450×10⁹/L, an absolute neutrophil count (ANC) (a type of white blood cell that fights infection) of at least 0.5×10⁹/L, and hemoglobin (the protein in red blood cells that carries oxygen) of at least 10g/dL.
  • Your peripheral blast count (an immature type of blood cell) must be less than 1%.
  • Your fibrosis score (a measure of scarring in the bone marrow) must be less than grade 2.
  • Your life expectancy must be more than 9 months.
  • You must be able to swallow oral medications (pills or liquids taken by mouth).

Who Cannot Join the Study?

  • You need blood transfusions, which means receiving packed red blood cells (a component of blood) more than twice a month for over three months, or having a hemoglobin level (a protein in red blood cells that carries oxygen) of 8g/dL or less in the 8 weeks before starting.
  • You have pulmonary hypertension, which is high blood pressure in the lungs, that requires you to use oxygen therapy.
  • You have congestive heart failure, a condition where the heart cannot pump blood well enough, classified as NYHA class 3 or 4 (severe stages where symptoms limit daily activity).
  • Your left ventricular ejection fraction (LVEF), which is a measurement of how much blood the heart pumps out with each beat, is less than 45% as shown by an echocardiogram (an ultrasound of the heart).
  • Your corrected QT interval (mQTcF), which is a measurement of the time it takes for your heart muscle to recharge between beats, is greater than 450ms.
  • You have an uncontrolled or untreated infection with HIV (a virus that attacks the immune system), hepatitis B, or hepatitis C (viruses that affect the liver).
  • You are currently using prohibited medications, such as cytotoxic agents (drugs that kill cells), hematopoietic growth factors (drugs that help make blood cells), or MAOIs (a type of antidepressant), or you are expected to need them.
  • You have a known hypersensitivity reaction, which is an allergic reaction, to drugs similar to MAOIs.
  • You have a history of gastrointestinal (GI) function issues, such as chronic diarrhea or having had gastric bypass surgery, that might prevent the body from absorbing medicine.
  • You have previously used another investigational agent (a drug being tested in a study) and have not finished a washout period, which is the time needed for a drug to leave your system.
  • Your condition has not responded to bomedemstat or other LSD-1 inhibitors (specific types of medicine used for this condition).
  • Your ECOG score, which is a scale used to measure how well a patient can perform daily activities, is 3 or higher.
  • You have a known sensitivity or allergy to iadademstat.
  • You have a known current drug or alcohol abuse.
  • You have another medical condition that the doctor believes significantly increases your risk for the study.
  • You are currently pregnant, breastfeeding, or plan to become pregnant or breastfeed during the study.
  • You are unable to follow the specific rules and steps of the study.
  • You receive live or live-attenuated vaccines, which are vaccines containing a weakened form of a germ.
  • You have a history of splenectomy, which is the surgical removal of the spleen.
  • You have had major surgery within the last 4 weeks or are still recovering from the side effects of a recent surgery.
  • You have unresolved toxicities, which are lingering harmful side effects, from previous medical treatments.
  • You show signs of an increased risk of bleeding, such as abnormal results in aPTT or INR (tests that measure how long it takes blood to clot), a history of thrombocytopenia (low platelet count), or a known bleeding disorder like hemophilia.
  • You have significant renal insufficiency (kidney problems) or hepatic insufficiency (liver problems), measured by levels of creatinine, AST, ALT, or bilirubin (substances that indicate how well the kidneys and liver are working).
  • You have a history of cirrhosis (scarring of the liver) or evidence of fibrosis (scar tissue in the liver).
  • You have had an uncontrolled active infection (bacterial, viral, fungal, or parasitic) within 72 hours before starting the study.
  • You have had a fever higher than 38.5°C in the week before the first dose of the study medication.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Jerez de la Frontera Jerez De La Frontera Spain

Other Sites

Site Name City Country Status
Hospital General Universitario Morales Meseguer Murcia Spain
Hospital Del Mar Barcelona Spain
El Hospital Universitario De Gran Canaria Dr. Negrin Las Palmas De Gran Canaria Spain
Hospital Clinic De Barcelona Barcelona Spain
University Clinical Hospital Virgen De La Arrixaca Murcia Spain
Universidade De Santiago De Compostela Santiago De Compostela Spain
Ifrhbmqf Cnncya Dovkzxrtemqpvopod L'hospitalet De Llobregat Spain
Hgvhdkyk Vbke djpslhvw Barcelona Spain

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

Trial status

Country Status Recruitment Start
Spain Spain
Recruiting
31.03.2026

Trial locations

Investigated drugs:

Iadademstat is an oral liquid medication being tested to see if it can help lower high platelet counts in adults with a blood disorder called essential thrombocythemia, especially for those who have not responded well to or cannot tolerate their current medicine.

Investigated diseases:

Essential Thrombocythemia – This is a rare blood disorder characterized by the overproduction of platelets in the bone marrow. Platelets are the cells that help the blood clot. In this condition, the body produces an abnormally high number of these cells. As the disease progresses, the elevated platelet count can persist over time. This increase occurs because the bone marrow cells undergo changes that cause them to produce too many platelets.

Trial ID:
2025-523864-19-00
Protocol code:
CL06-ORY-1001
Trial Phase:
Therapeutic exploratory (Phase II)

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