Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study of Gandotinib for Patients with Myeloproliferative Neoplasms, Myelofibrosis, Essential Thrombocythemia, or Polycythemia Vera

Fast replyInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a group of blood cancers known as myeloproliferative neoplasms (MPNs). These include specific conditions such as myelofibrosis, essential thrombocythemia, and polycythemia vera. The treatment being tested in this study is a medication called gandotinib, which is also known by its code name LY2784544. The purpose of the study is to evaluate how effective this medication is in treating these blood cancers.

Participants in the study will take the medication in the form of a capsule, which is taken by mouth once a day. The study will monitor the response to the treatment over a period of time to see how well it works in managing the conditions. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment. The study aims to gather information on how the medication affects the body and its potential side effects.

By participating in this study, researchers hope to better understand the effectiveness of gandotinib in treating myeloproliferative neoplasms and to explore its potential as a treatment option for patients with these conditions. The study will help determine if this medication can improve the quality of life for those affected by these blood cancers.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, ensure that all previous therapies for myeloproliferative neoplasms (MPNs) have been discontinued for at least 14 days. This includes chemotherapy, immunomodulating therapy, immunosuppressive therapy, radiotherapy, and certain growth factors. Hydroxyurea and low-dose aspirin are allowed if maintained on a stable dose.

    Confirm the ability to swallow capsules and agree to use medically approved contraceptive precautions during the study and for 3 months after the last dose. Females must have a negative pregnancy test within 7 days before the first dose and must not be breastfeeding.

  2. Step 2

    Medication administration

    The study involves taking a medication called LY2784544, which is administered in the form of a capsule. This medication is taken orally once daily.

    The primary goal is to assess the activity of LY2784544 in patients with MPNs, including polycythemia vera, essential thrombocythemia, and myelofibrosis.

  3. Step 3

    Monitoring and assessments

    Throughout the study, regular monitoring and assessments will be conducted to evaluate the response to the medication. This includes measuring the objective response rate and characterizing the toxicity profile.

    Patients will be assessed for efficacy and response criteria, ensuring that the treatment is working as intended.

  4. Step 4

    Completion of the study

    The study is estimated to end by December 31, 2024. Participation will continue until the study's conclusion or until it is determined that the treatment is no longer beneficial.

    Upon completion, follow-up procedures may be conducted to ensure the patient's well-being and to gather final data for the study.

Who can join the trial?

15 criteria

  • Have a diagnosis of polycythemia vera (PV), essential thrombocythemia (ET), or myelofibrosis (MF) as defined by the World Health Organization (WHO).
  • Have stopped all previous approved treatments for myeloproliferative neoplasms (MPNs) for at least 14 days and recovered from any immediate effects of those treatments. You can continue using hydroxyurea if your dose has been stable for at least 4 weeks. Low-dose aspirin is also allowed.
  • Are reliable and willing to be available for the entire study and follow the study procedures.
  • If you are a male or female who can have children, you must agree to use medically approved birth control during the study and for 3 months after the last dose of the study drug.
  • If you are a female who can have children, you must have a negative pregnancy test within 7 days before the first dose of the study drug and must not be breastfeeding.
  • Are able to swallow capsules.
  • If you have had recent major surgery, at least 28 days must have passed since the surgery, and you must have recovered well according to your doctor.
  • If you are enrolling in Cohort 12, you must have shown intolerance to ruxolitinib, failure to respond to it, or disease progression while on it.
  • Have failed or are intolerant of standard therapies or refuse to take standard medications.
  • Have intermediate 1, intermediate 2, or high-risk MF according to the Dynamic International Prognostic Scoring System (DIPPS Plus) for Primary Myelofibrosis, or have symptomatic MF with a spleen larger than 10 cm below the left rib margin, or have post-polycythemic MF, or have post-ET MF.
  • Have a measurable JAK2 V617F mutation, unless you are in Cohorts 10 and 11, where you must be negative for this mutation.
  • Are 18 years of age or older.
  • Have given written informed consent before any study-specific procedures.
  • Have adequate organ function.
  • Have a performance status of 0, 1, or 2 on the Eastern Cooperative Oncology Group (ECOG) scale, which measures your ability to perform daily activities.

Who cannot join the trial?

9 criteria

  • Patients with other types of cancer that are not related to myeloproliferative neoplasms (MPNs), which include myelofibrosis, essential thrombocythemia, and polycythemia vera.
  • Patients who have had a heart attack or stroke in the past 6 months.
  • Patients with uncontrolled high blood pressure.
  • Patients with severe liver or kidney disease.
  • Patients who are pregnant or breastfeeding.
  • Patients who are participating in another clinical trial.
  • Patients who have an allergy to the study medication.
  • Patients with a history of drug or alcohol abuse in the past year.
  • Patients with any other medical condition that the study doctors think would make it unsafe for them to participate.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

LY2784544 is a medication being studied for its potential to help people with certain blood disorders known as myeloproliferative neoplasms. These disorders include conditions like polycythemia vera, essential thrombocythemia, and myelofibrosis. The medication is taken once a day, and the main goal of the study is to see how well it works in improving the symptoms of these conditions. By taking this medication, researchers hope to find out if it can help reduce the number of abnormal blood cells and improve overall health in patients with these disorders.

What is already known about the treatment

LY2784544 – LY2784544 is a medication currently being studied in clinical trials for its potential use in treating myeloproliferative neoplasms, including myelofibrosis, essential thrombocythemia, and polycythemia vera. It is administered orally, meaning patients take it by mouth, typically once a day. The drug is still under investigation, so it is not yet widely available or fully endorsed in medical literature. LY2784544 works by targeting specific pathways in the body that contribute to the overproduction of blood cells, which is a hallmark of these conditions. It belongs to a class of drugs known as kinase inhibitors, which help regulate cell growth and division.

Investigated diseases

  • Myeloproliferative Neoplasms (MPNs)

    Myeloproliferative neoplasms are a group of diseases where bone marrow cells that produce blood cells develop and function abnormally. These conditions lead to an overproduction of one or more types of blood cells, including red blood cells, white blood cells, or platelets. The disease progresses as the abnormal cells accumulate in the blood and bone marrow, potentially causing various symptoms and complications. Over time, MPNs can lead to changes in blood cell counts and may affect the spleen and other organs. The progression can vary significantly among individuals, with some experiencing slow changes and others more rapid developments.

  • Myelofibrosis

    Myelofibrosis is a type of myeloproliferative neoplasm characterized by the replacement of bone marrow with fibrous tissue. This process disrupts the normal production of blood cells, leading to anemia, fatigue, and an enlarged spleen. As the disease progresses, the bone marrow becomes increasingly fibrotic, further impairing blood cell production. Patients may experience symptoms related to low blood counts and spleen enlargement. The progression of myelofibrosis can vary, with some individuals experiencing stable disease and others facing more rapid changes.

  • Essential Thrombocythemia

    Essential thrombocythemia is a myeloproliferative neoplasm where the bone marrow produces too many platelets. This overproduction can lead to abnormal blood clotting or bleeding. The disease progresses as the high platelet count persists, potentially causing complications such as blood clots or bleeding episodes. Some individuals may experience symptoms like headaches or dizziness, while others remain asymptomatic. The progression of essential thrombocythemia can vary, with some patients maintaining stable platelet levels and others experiencing changes over time.

  • Polycythemia Vera

    Polycythemia vera is a myeloproliferative neoplasm characterized by the overproduction of red blood cells. This increase in red blood cells thickens the blood, potentially leading to complications such as blood clots. As the disease progresses, patients may experience symptoms like headaches, dizziness, and an enlarged spleen. The thickened blood can affect circulation and lead to various symptoms and complications. The progression of polycythemia vera can vary, with some individuals experiencing stable disease and others facing more rapid changes.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-514254-69-00Protocol codeI3X-MC-JHTBEstimated enrolment7 patientsSponsorEli Lilly & Co.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).