Active clinical trials for "Primary Myelofibrosis"

Myeloproliferative neoplasms (MPNs) are blood disorders that occur when the body makes too many white or red blood cells, or platelets. This overproduction of blood cells in the bone marrow can create problems for blood flow and lead to various symptoms. One of the major problems is the formation of blood clots. These may form in the veins of a patient's legs or arms where they cause leg or arm pain, swelling or difficulty walking. These clots may travel to the lung and then cause chest pain, shortness of breath and sometimes death. Blood clots can also lead to poor or no blood flow to one's heart, brain, or other organs, causing damages that cannot be easily or ever repaired, such as stroke or heart attack. Patients diagnosed with certain types of MPN are associated with a higher risk of developing blood clots and related complications. For this reason, MPN patients are usually treated with low-dose aspirin, a common drug used for blood clot prevention, on long-term basis to prevent the formation of blood clots and other complications. However, recent studies also show that the risk of blood clots remains elevated in MPN patients treated with aspirin, and there may not be improvement or reduction in fatal or other events that are associated with blood clots. In addition, since this medical condition is rare, so there's a lack of studies done with high quality results to help physicians decide the best treatment plan for these patients. The study drug, apixaban, is a new type of orally-taken blood thinner that has been shown to be effective and safe for prevention and treatment of blood clots in various patient populations. The investigators will evaluate whether apixaban is safer and/or better at preventing blood clots and other complications in MPN patients compared to aspirin.

The only curative treatment in patients with primary or secondary myelofibrosis is allogeneic hematopoietic stem cells (HSCT). It has been reported that intermediate and higher risk patients according to international prognostic scores benefit from HSCT in terms of survival (Kröger et al, 2015). In 2013, we conducted in France a prospective trial testing the use of ruxolitinib before transplantation ("JAK-ALLO study" NCT01795677). Outcome of patients was better in patients transplanted with a matched sibling donor than an unrelated donor confirming other studies (Kröger et al, 2009; Rondelli et al, 2014). In the JAK-ALLO trial, acute GVHD incidence was high, often hyperacute and severe. Recently, the EBMT group has reported a registry study on familial haplo-identical transplantation (haplo) in patients with myelofibrosis (Raj et al, 2018). Post-transplant cyclophosphamide was used in 59% of cases. One-year overall survival (OS) and disease-free survival (DFS) were 61 and 58% which favorably compared to outcome after unrelated transplantation. Genova team has also reported impressive results after haplo-identical transplantation in their center (Bregante et al, 2015). Bregante et al have reported outcome of 2 cohorts transplanted from 2000 to 2010 and from 2011 to 2014. The main difference between the 2 periods is the more frequent use of haplo in the second period (54% versus 5%). Outcome was much better in the second period with OS at 70% versus 49% and authors suggest that this improvement is related to the best outcome among haplo transplantation. The improvement of outcome after haplo has been attributed to a better GVHD prophylaxis, especially with the use of post-transplant cyclophosphamide. Given the poor outcome after unrelated transplantation and especially in HLA mismatched unrelated setting and encouraging results in family haplo identical transplantation, this current study proposes to test haplo-identical transplantation in myelofibrosis patients without a matched related donor. The main objective of this study is disease and rejection-free survival one year after haplo-identical transplantation in patients with primary or secondary myelofibrosis.

The purpose of this study is to assess the real-world safety of fedratinib for the treatment of adult participants with primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), or post essential thrombocythemia myelofibrosis (post-ET MF) who were previously treated with ruxolitinib. Participants will represent the overall patient population with PMF, post-PV MF or post-ET MF who lost adequate response to and/or are intolerant to ruxolitinib. Inadequate response definitions will follow Ministry of Food and Drug Safety-approved label and reimbursement criteria of the Health Insurance Review & Assessment Service.

The main purpose of this Phase 2 study is to evaluate the efficacy of selinexor in participants with myelofibrosis (MF) and moderate thrombocytopenia based on spleen volume reduction (SVR). Additional efficacy parameters and safety will also be assessed during the study.

The purpose of this research study is to see how effective hematopoietic stem cell transplantation (HCT) is compared to best available non-transplant therapies (BAT) in patients with high risk myelofibrosis. This will be done by asking participants to choose the treatment that they prefer to receive (HCT or BAT) and then comparing the outcomes of the participants in both treatment groups.

Patients with graft failure or delayed engraftment may benefit from a hematopoietic stem cell boost or an additional hematopoietic stem cell transplantation procedure. In such settings standard immune suppression strategies are avoided due to their myelosuppressive nature. Therefore those patients are at increased risk of graft versus host disease, and the infusion of a CD34 selected graft would reduce such a risk. The infusion of CD34 selected graft using CliniMACS plus is currently FDA FDA-approved indication for acute myeloid leukemia. However, the use of the Prodigy would streamline the processing, in terms of hands-off procedure, allowing to provision of this product to the patients without strains on the cell therapy lab team. This procedure has been demonstrated safe and effective in several single-center studies and is currently in advanced phase investigation in several studies for malignant and non-malignant conditions.

All patients diagnosed or followed in Brest University Hospital for Philadelphia negative myeloproliferative neoplasms will be included in this observational study. Myeloproliferative neoplasms recorded included: polycythemia vera, essential Thrombocythemia and Primary Myelofibrosis. This is a not interventional study. Alive patients need to sign a non-opposition consent form. Patients will be followed until last news (death, change of reference centre...).

The mandate of this MPN registry is to collect clinical information, including molecular results, from consenting patients with a variety of MPNs at different time points during the course of their disease.

This randomized phase I trial studies the side effects of vaccine therapy in preventing cytomegalovirus (CMV) infection in patients with hematological malignancies undergoing donor stem cell transplant. Vaccines made from a tetanus-CMV peptide or antigen may help the body build an effective immune response and prevent or delay the recurrence of CMV infection in patients undergoing donor stem cell transplant for hematological malignancies.

This is a global, multicenter, open-label, repeat-dose, Phase 1/2 study consisting of a Dose Escalation Phase (Phase 1) and a Cohort Expansion Phase (Phase 2). In both phases, KB004 will be administered by IV infusion once weekly as part of a 21-day dosing cycle.