Essential thrombocythaemia (ET) and polycythaemia vera (PV)
TAMARIN Effects of TAMoxifen on the Mutant Allele Burden and Disease Course in Patients with Myeloproliferative Neoplasms – ET/PV and MF
The TAMARIN study is a very exciting study based on UK science and is now open in 13 hospitals across the UK. The study is a multicentre phase II clinical trial which will assess the safety and activity of Tamoxifen in MPNs. More information on this trial can be found here…
Recruitment start date: 26/08/16 Recruitment end date: 31/12/18. Preliminary results are very exciting and recruitment to the trial is now closed. We hope to add the results of the trial when they are available.
GIVINOSTAT currently being evaluated in patients with PV.
For further details of this study, including inclusion and exclusion criteria click here… Study end date: June 2020 This trial is active but no longer recruiting.
KRT-232-101 Phase 2 Clinical Trial KRT-232-101 is a Phase 2a-2b, global, open-label study of KRT-232 in patients with Primary Myelofibrosis, post-Polycythaemia Vera Myelofibrosis, or post-Essential Thrombocythaemia Myelofibrosis who have failed previous treatment with a JAK inhibitor and in patients with phlebotomy-dependent Polycythaemia Vera.
In PART A, patients must be resistant or intolerant to Hydroxyurea (Hydroxycarbamide) or have undergone treatment with interferon. In PART B, patients must be resistant or intolerant to Hydroxyurea (Hydroxycarbamide).
Locations running this trial in the UK are: Birmingham NHS Foundation Trust; Greater Glasgow and Clyde NHS Foundation Trust; Oxford University Hospital NHS Foundation Trust. There are also a number of locations in the USA, details are available on the link below.
Full details for the study can be found on the ClinicalTrials.gov website.
Myelofibrosis (MF)
IMpactMF Study
A Phase 3 Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor Treatment. The purpose of the study is to evaluate the overall survival of participants treated with imetelstat compared to best available therapy with intermediate-2 or high-risk Myelofibrosis (MF) who are refractory to Janus Kinase (JAK)-Inhibitor treatment. Further details about this study, including locations, can be found here. Please note that 4 UK trial sites have confirmed participation in the trial: Guy’s & St Thomas’ Hospital, London – Lead Prof Claire Harrison; Maidstone Hospital, Kent – Lead Dr Evangelia Dimitriadou; Oxford University Hospital, Oxford – Lead Prof Adam Mead; St Bartholomew’s Hospital, London – Lead Dr Heather Oakervee.
TAMARIN Effects of TAMoxifen on the Mutant Allele Burden and Disease Course in Patients with Myeloproliferative Neoplasms – ET/PV and MF
The TAMARIN study is a very exciting study based on UK science and is now open in 13 hospitals across the UK. The study is a multicentre phase II clinical trial which will assess the safety and activity of Tamoxifen in MPNs. More information on this trial can be found here…
Recruitment to this trial is now closed, we hope to add the results when they are available.
STUDY OF THE SAFETY OF PIM447 in combination with Ruxolitinib (INC424) and LEE011 in patients with Myelofibrosis.
For further details of this study, including inclusion and exclusion criteria click here…
NAVITOCLAX A Phase 2 Single-Arm, Open-Label Study Evaluating Tolerability and Efficacy of Navitoclax in Combination with Ruxolitinib in Subjects with Myelofibrosis. Navitoclax is an experimental drug that kills cancer cells by blocking a protein that allows cancer cells to stay alive. It has been shown to have an effect on cells that cause MF.
For further details about this study including inclusion and exclusion criteria click here… (estimated) Primary completion date: 26 February 2022. (estimated) Study end date: 2 February 2029
KRT-232-101 Phase 2 Clinical Trial
KRT-232-101 is a Phase 2a-2b, global, open-label study of KRT-232 in patients with Primary Myelofibrosis, post-Polycythaemia Vera Myelofibrosis, or post-Essential Thrombocythaemia Myelofibrosis who have failed previous treatment with a JAK inhibitor and in patients with phlebotomy-dependent Polycythaemia Vera.
In PART A, patients must be resistant or intolerant to Hydroxyurea (Hydroxycarbamide) or have undergone treatment with interferon. In PART B, patients must be resistant or intolerant to Hydroxyurea (Hydroxycarbamide).
Locations running this trial in the UK are: Birmingham NHS Foundation Trust; Greater Glasgow and Clyde NHS Foundation Trust; Oxford University Hospital NHS Foundation Trust. There are also a number of locations in the USA, details are available on the link below.
Full details for the study can be found on the ClinicalTrials.gov website.
MOMENTUM clinical trial for people with Myelofibrosis (MF)
The MOMENTUM Clinical Trial for Patients with Myelofibrosis will evaluate the effectiveness and safety of the investigational drug, momelotinib, compared to another drug (danazol), in treating myelofibrosis. Patients who participate in the clinical trial will randomly be assigned so that 120 patients receive momelotinib and 60 patients receive danazol, for a total of 180 patients. Momelotinib is a type of drug known as a Janus kinase (or JAK) inhibitor which is designed to address the three major problems of myelofibrosis including the debilitating symptoms; anaemia (low red blood cell counts), or other low blood cell counts; and an enlarged spleen. Because momelotinib inhibits the JAK enzymes and another enzyme target involved in red blood cell production, called ACVR1, it may be able to treat myelofibrosis symptoms and enlarged spleen while also increasing red blood cell numbers, improving anaemia. This profile makes momelotinib different to other JAK inhibitors currently available which do not treat anaemia and usually make it worse. Momelotinib treatment will be compared to that of danazol which is a drug listed as a potential treatment option for myelofibrosis patients with anaemia. It works in a completely different way to momelotinib and is not a JAK inhibitor. Further details about this trial can be found here, if you would like to take part and fit the criteria, please discuss this with your consultant haematologist.
IMG-7289 in patients with Myelofibrosis (MF)
A Multi-Center, Open Label Study to Assess the Safety, Steady-State Pharmacokinetics and Pharmacodynamics of IMG-7289 in Patients with Myelofibrosis. Eligibility Criteria:- Age 18 or over and have an ECOG performance score of 2 or less. Diagnosis of either primary myelofibrosis (PMF) per World Health Organization criteria, post-polycythaemia vera myelofibrosis (PPV-MF), or post-essential thrombocythaemia myelofibrosis (PET-MF) and meet the trial criteria. This study is currently recruiting, full details can be found here including locations, which in the UK include Guy’s Hospital, London, The Churchill Hospital, Oxford, Heartlands Hospital, Birmingham and The Royal Bournemouth Hospital, Bournemouth, if you would like to take part in this study, please discuss this with your consultant haematologist. Primary completion date: 31 Dec 20, estimated study completion date: 01 Feb 21.
PATIENTS WITH AGGRESSIVE MPN or leukaemia after MPN
PHAZAR – a study for patients developing leukaemia after MPN or patients whose disease is close to leukaemia is currently open in 13 centres. This study is assessing the role of combining a standard leukaemia drug azacytidine with Ruxolitinib.
For further details of this study, including inclusion and exclusion criteria click here…
Study Data
We are currently awaiting data from a large number of studies that were open including:
SIMPLIFY 1 & 2
IMBARK
PROMOTE
Results will be published when they are available
SYMPTOM ASSESSMENT
THE MEASURES STUDY a symptom assessment study is open in over 20 centres in the UK.
Myeloproliferative neoplasms (MPNs) are associated with a range of symptoms that can affect many different systems of the body. We have developed a diary designed to record the impact of symptoms in patients with MPNs. It is based on a previously tested questionnaire, the MPN symptom assessment form (MPN-SAF). This study aims to test the diary to see if it is effective at recording the impact of patients symptoms.
To read more about this study click here…
SAMPLE BANKS
Studies with banks in Cambridge, Salisbury, Oxford and Guy’s Hospital are open.
The project will sequence 100,000 genomes from around 70,000 people. Participants are NHS patients with a rare disease, plus their families, and patients with cancer, MPNs are included in the 100k genome project.
The aim is to create a new genomic medicine service for the NHS – transforming the way people are cared for. Patients may be offered a diagnosis where there wasn’t one before. In time, there is the potential of new and more effective treatments.
For further details of this study click here…
Useful websites
Visit any of these websites to find more information about trials.
If you are interested in enrolling in a trial and live in the U.K. please email us at info@mpnvoice.org.uk for details of the centres/hospitals running the trials, please tell us where you live and which hospital you attend. If you live outside the U.K. please contact your haematologist for details.
In the United States
You can learn more about trials from several organisations in the United States.
- MPN Research Foundation (US) The MPN Research Foundation (US) raises funds for MPD research and reports on clinical trials, press releases from biotech and pharmaceutical companies and the latest discoveries.
- NIH The National Institutes of Health In the US offers detailed and accurate information on diseases and a search tool to find clinical trials internationally.
- NCI The National Cancer Institute (NCI)’s clinical trials page offers detailed information on trials taking place in the UK and US – search by disease name, for instance “myelofibrosis”.
You can also watch the video interview of Albert, who enrolled on a JAK2 inhibitor trial. You can read about people who are taking the newest drugs in Real Stories.
