Active clinical trials for "Primary Myelofibrosis"

Recently, it has been demonstrated that iron overload is associated with the appearance of labile plasma iron (LPI). LPI is redox active and is rapidly taken up by cells, leading to a rise in the labile iron pool (LIP) and catalyzing generation of reactive oxygen species (ROS), which can lead to cellular damage. The LPI data are mostly derived from thalassemia iron overload research , however, there are a few data describing LPI and its correlations with the classical iron overload parameters (ferritin, TSAT) in acute anemias such as MDS Therefore we are going to assess LPI in iron overloaded myelodysplastic syndromes (MDS) (low and high risk) and primary myelofibrosis, in order to assess whether it can be used as alternative to the routinely used parameters; TSAT and ferritin levels.

Compassionate use access to Momelotinib/GSK3070785 for eligible participant with diagnosis of intermediate or high-risk myelofibrosis (MF), including primary myelofibrosis (PMF) or secondary myelofibrosis (post- polycythemia vera/ essential thrombocytopenia (PV/ET)), with anemia.

This is an expanded access program (EAP) for eligible participants designed to provide access to fedratinib. Expanded access is only available in markets where fedratinib is not yet approved.

The purpose of this Cohort Treatment Plan is to allow access to ruxolitinib for eligible patients diagnosed with PMF, PPV MF or PET MF. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. The requesting Treating Physician submitted a request for access to drug (often referred to as Compassionate Use) to Novartis which was reviewed and approved by the medical team experienced with the drug and indication.

There is a paucity of data on the aetiology of myeloproliferative neoplasms (MPNs). The investigators conducted a systematic review of the literature which identified several cohort and case-control studies that have investigated a wide range of potential medical, environmental and occupational risk factors. However, these studies have been limited by a wide variation in case definition and small sample sizes limiting the potential to detect modest risk differences between cases and controls. The research group propose an exploratory case-control study of 100 patients with classic MPNs and 200 controls to determine the optimal methods for roll out of this study to a multi-centred UK-based case-control study that will investigate the aetiology of MPN subtypes. The objectives of the study are to evaluate recruitment procedures, response rates, the development of a telephone administered questionnaire, compare occupational exposure assessment using OccIDEAS, a novel web based program, with a job-exposure matrix (FINJEM), evaluate the feasibility of collection of DNA from saliva compared to blood samples, identify potential aetiologic factors associated with MPNs and explore assessment of quality of life. The findings of this exploratory study will form the basis of a protocol for a large United Kingdom (UK)-wide case-control study of MPNs.