Myelofibrosis with Myeloid Metaplasia

Clinical Trials

Below is a list of Myelofibrosis with Myeloid Metaplasia clinical trials from the clinical trials database at Mayo Clinic.

This list includes only trials about which Mayo researchers choose to publish information. Mayo Clinic may be conducting other trials which are not in this database. Mayo's clinical trials include experimental treatments, often unavailable elsewhere, which frequently lead to improved patient care for people worldwide. Patients should ask their doctor at Mayo about clinical trials appropriate for their situation.

A Phase 1/2, Open-Label Study of the JAK2 Inhibitor INCB018424 Administered Orally to Patients with Primary Myelofibrosis (PMF) and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)
Patients are being asked to take part in this research study because they have been diagnosed with primary myelofibrosis (PMF, normal bone marrow cells have been replaced by fibrous tissue) and post polycythemia vera/essential thrombocythemia myelofibrosis (Post-PV/ET MF, there are excessive red blood cells and/or excessive platelets). These are myeloproliferative disorders (MPDs).

The study will evaluate the safety, tolerability, side effects, levels of the drug in a patient's blood, and the maximum tolerated dose of the investigational drug (INCB018424). Investigational means that the drug being tested has not been approved by the United States Food and Drug Administration (FDA) as a prescription or over-the-counter medicine. Information from this study will be used to determine how well the study medication works and to have a better understanding of how the study medication works in people with a different genetic mutation (change), such as JAK2. Study of the structure of normal and abnormal chromosome (genetic) material and JAK2 mutation test
will be done during the course of the patient's participation in the study. These are genetic tests that will require a blood draw or bone marrow aspirates or biopsies.

Human cells contain genetic material that acts as an instruction book to direct cell
splitting and copying, to help form different body parts, and to determine our physical appearance. This genetic material (DNA and RNA) is organized in a code that produces
proteins that perform many bodily functions. Parts of the genetic code that code into specific proteins are called genes. Differences in the gene's code could mean a different eye color, different skin color, or in some cases differences in how people get certain diseases or why their bodies react differently to certain medications. Studying the difference in genes can lead to better understanding of diseases and body processes, and can help develop new medicines.

There will be 2 phases of the study. The purpose of the dose-escalation phase is to find out how safe it is, what the highest dosage of the drug is that can be tolerated, how the drug is absorbed and its action on the patient's body, the effectiveness and to determine a dose of INCB018424 for the expanded cohort phase of the study. The purpose of the expanded cohort is to enroll more patients to further assess the safety and usefulness of the study medication at one specific dose. Depending on the time of the patient's enrollment, they will participate in either the dose-escalation or expanded cohort phase of the study. Patients will be told which phase of the study they are in.
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Comparison of Peripheral Blood Stem Cell Transplantation with Bone Marrow Transplantation for the Treatment of Serious Hematological Malignancies
This research study will compare the outcomes of study participants who have had peripheral blood stem cell transplantation from unrelated donors with those who have had bone marrow transplant from unrelated donors for the treatment of serious hematological malignancies.

Two types of stem cell transplantation have been used to treat patients with certain types of serious diseases. Stem cells can be obtained from bone marrow or from circulating blood (peripheral blood stem cells). Participants may be treated with a transplant of either bone marrow or peripheral blood stem cells (PBSC) from unrelated donors.

Both of these types of transplant have been successful for the treatment of leukemia and myelodysplasia. The goal of this research study is to see if there are better results using a bone marrow transplant or a PBSC transplant from unrelated donors.
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MC0788, "Phase II Trial of Low Dose Decitabine (Dacogen) in Patients with Primary Myelofibrosis and Post ET/PV Myelofibrosis"
Patients are being asked to take part in this research study because they have myelofibrosis, either by itself or associated with polycythemia vera or essential thrombocytopenia. This study is being done to test a new drug called decitabine to see what effects (good and bad) it has on the patient and their disease. Decitabine has been approved by the U.S. Food and Drug Administration (FDA) for treatment of a related disease called myelodysplastic syndrome, but not myelofibrosis.
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