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

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What is this study about?

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.

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 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 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 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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Medical University Of Vienna	Vienna	Austria

Other Sites

Site Name	City	Country	Status
Johannes Wesling Klinikum Minden	Minden	Germany
Fintfaulc Pwhg Lo Ipquqwlpupett Bwdiljwdd Dkh Hoflzyhe Uoybvwgzjzpxv La Pyq	Madrid	Spain

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	22.05.2012
Germany	Not recruiting	22.05.2012
Spain	Not recruiting	22.05.2012

Trial locations

Investigated drugs:

Gandotinib

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.

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 ID:

2024-514254-69-00

Protocol code:

I3X-MC-JHTB

NCT ID:

NCT01594723

Trial Phase:

Therapeutic exploratory (Phase II)

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

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