Trials for Essential Thrombocythaemia (ET)
Awaiting information for trials for Essential Thrombocythaemia – this page will be updated in due course.
Trials for Polycythaemia Vera (PV)
Name: MITHRIDATE
Title: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High-Risk Polycythaemia Vera (MITHRIDATE)
Summary: A phase III randomised trial in high-risk PV consisting of ruxolitinib versus best available therapy, where best available therapy is a choice of interferon alpha, any formulation permitted (IFN) or hydroxycarbamide (HC), and which will be chosen by your doctor.
In terms of this study, you have high risk PV if you have, or have had, a high white blood cell count (>11 x 109/L) and at least one of the following:
- are over 60 years old
- have had a blood clot or bleeding problem
- have, or have had, a platelet count more than 1000 x 109/L
- Or diabetes or high blood pressure needing treatment.
You must have been diagnosed in the past 15 years and if you had already had a drug treatment have received it for less than 10 years.
Ruxolitinib will be compared to Interferon and Hydroxycarbamide to understand if in high-risk PV it shows any benefits over the standard treatments, whether side effects occur and how it affects quality of life.
UK Chief Investigator: Professor Claire Harrison
Main Eligibility – Who can’t take part
You cannot join this trial if any of these apply.
You:
- have had both hydroxycarbamide and interferon alpha
- stopped treatment because it wasn’t working or the side effects were very bad
- have already had ruxolitinib
- have myelofibrosis
- have a low count of platelets and, or a type of white blood cell called neutrophils
- have had heart problems such as a heart attack in the past six months, an irregular heartbeat or angina that isn’t controlled by medication
- have an active infection including hepatitis B, hepatitis C or tuberculosis (TB)
are pregnant or breastfeeding
Sites: Open in almost 40 UK sites
Other: Clinical teams at participating hospitals will invite patients they deem eligible but please feel free to discuss with them.
Name: VERIFY
Title: A Phase 3 Study of Rusfertide in Patients With Polycythaemia Vera (VERIFY)
Summary: The purpose of the VERIFY clinical trial is to evaluate how safe and effective rusfertide is in maintaining haematocrit control and improving PV symptoms.
Rusfertide, also known as PTG-300, is an investigational drug designed to slow the release of iron into the blood. The body needs iron to make red blood cells. With less iron in the blood, the body is not able to make as many red blood cells. As a result, rusfertide may help keep haematocrit levels under control. It is given as a subcutaneous, (below skin), injection. This is a Phase 3 study in patients with PV with three parts overall.
Part 1a: randomised, placebo control add on. Patients will be randomised 1:1 between 32 weeks of add-on rusfertide or placebo treatment. Rusfertide or placebo will be ‘added-on’ to each subject’s ongoing therapy for PV. The patient and the clinical team will be blinded to whether the patient is getting rusfertide or a placebo.
Part 1b: all patients who complete part 1A successfully will receive rusfertide for 20 weeks (Week 32 through Week 52).
Part 2: long term extension for 104 weeks during which all subjects who complete Part 1b will continue to receive rusfertide. Approximately 250 participants will take part in this study at approximately 100 study sites globally. Enrolment will be competitive globally.
UK Chief Investigator: Professor Ciro Rinaldi
Main Eligibility If you have PV and have received at least 3 venesections in the last 6 months or at least 5 within in the last year for inadequate haematocrit control, you may qualify.
Sites: There are currently six research sites in the UK:
Pilgrim Hospital, Lincoln, Boston
University College Hospital, London
Royal Free Hospital, London; including Barnet Hospital
Northwick Park Hospital, Harrow, London
Churchill Hospital, Oxford
The Royal London Hospital, London
Other: Clinical teams at participating hospitals will invite patients they deem eligible but please feel free to discuss with them.
Name: Imprssion
Title: A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients with Polycythaemia Vera
Summary: Sapablursen is an investigational antisense medicine being studied as a potential treatment for PV. Sapablursen is designed to reduce the production of transmembrane protease (also known as TMPRSS6), a protein produced in the liver. Inhibition of this protein leads to increased production of hepcidin, which reduces iron availability and results in reduced red blood cell production (erythropoiesis) in the bone marrow. Eligible participants will be assigned to receive one of 2 dosing levels: a higher or lower dose level of sapablursen. They will have an equal chance of being assigned to either dose level during the treatment period, which lasts about 16 months plus a 4-week post-last dose visit. They will be blinded to the high or the low dose level. Everyone will receive drug, there is no placebo.
UK Chief Investigator: Professor Ciro Rinaldi
Main Eligibility: You may be eligible to participate in the IMPRSSION clinical trial if you:
Have been clinically diagnosed with polycythaemia vera (PV)
Have approximately six phlebotomies a year
Are between 18 years and 90 years of age
Are on a stable dose or are not taking cytoreductive therapy
Conditions that may exclude individuals from study participation include:
Known primary or secondary immunodeficiency
Post-polycythaemia vera myelofibrosis
Sites: Lincoln County Hospital
Churchill Hospital, London
University College Hospital, London
Other: Clinical teams at participating hospitals will invite patients they deem eligible but please feel free to discuss with them.
Trials for Myelofibrosis (MF)
Name: PROMise
Title: A trial of PLX2853 with ruxolitinib for myelofibrosis (PROMise)
Summary: PLX 2853 is a new targeted drug that is also a cancer growth blocker. It is a BET inhibitor (BETi) which blocks certain activities in the cancer cells that they need to grow and divide. In this trial you continue to take ruxolitinib as usual and you also take PLX2853. This phase I Trial will evaluate the combination of a Plexxikon BETi with ruxolitinib in patients with high or intermediate-2 risk myelofibrosis not receiving an adequate response with ruxolitinib alone. Up to 60 patients will take part in this trial nationally.
The aims of this trial are hence to find out:
- the best dose of PLX2853 to take with ruxolitinib
- how well the combination works to reduce the size of the spleen
the side effects and how it affects quality of life
UK Chief Investigator: Professor Adam Mead
- Main Eligibility are aged 16 years or above and have myelofibrosis that is intermediate risk 2 or high risk.
- have had at least 24 weeks of ruxolitinib and your spleen is more than 5cm from the bottom of your chest (ribcage). Your doctor will know this.
- have satisfactory blood test results with platelets >75×109/L and neutrophils >1×109/L
- <10% blasts in peripheral blood and/or bone marrow
- receiving the same dose of ruxolitinib for at least 4 weeks before joining the trial
have adequate kidney and liver function
Sites: St James’s University Hospital
University College London Hospital
Royal Hallamshire Hospital, Sheffield
Churchill Hospital, Oxford
Southampton General Hospital
University Hospital of Wales
Belfast City Hospital
Nottingham City Hospital
Guy’s and St Thomas’ NHS FT
Beatson West of Scotland Cancer Centre
Other: Clinical teams at participating hospitals will invite patients they deem eligible but please feel free to discuss with them.
Name: PACIFICA
Title: A Phase 3 Study of Pacritinib in Patients with Primary Myelofibrosis, Post Polycythaemia Vera Myelofibrosis, or Post-Essential Thrombocythaemia Myelofibrosis (PACIFICA)
Summary: This study is evaluating 200 mg twice daily of the JAK inhibitor pacritinib compared to your physician’s choice (P/C) therapy in patients with Myelofibrosis and severe thrombocytopaenia (platelet count <50,000/μL).
Primary Objectives:
To compare the efficacy of pacritinib with that of physician’s choice (P/C) therapy, as assessed by the proportion of patients achieving a ≥35% spleen volume reduction (SVR) from baseline at 6 months
To compare the efficacy of pacritinib with P/C therapy, as assessed by the proportion of patients achieving a ≥50% reduction in Total Symptom Score (TSS) from baseline at 6 months
UK Chief Investigator: Professor Claire Harrison
Main Eligibility Diagnosis and Inclusion Criteria
- Primary MF, post-polycythaemia vera MF, or post-essential thrombocythemia MF
- Platelet count of <50,000/μL at Screening (Day -35 to Day -3)
- Dynamic International Prognostic Scoring System Intermediate-1, Intermediate-2, or High-Risk
- Palpable splenomegaly ≥5 cm below the lower costal margin (LCM) in the midclavicular line as assessed by physical examination
- TSS of ≥10 on the MPN-SAF TSS 2.0 or a single symptom score of ≥5 or two symptoms of ≥3, including only the symptoms of left upper quadrant pain, bone pain, itching, or night sweats. The TSS criteria need only to be met on a single day.
- Age ≥18 years
- Eastern Cooperative Oncology Group performance status 0 to 2
- Peripheral blast count of <10% throughout the Screening period prior to randomization
- Absolute neutrophil count of ≥500/µL
- Left ventricular cardiac ejection fraction of ≥50% by echocardiogram or multigated acquisition scan
- Adequate liver and renal function
- Adequate coagulation
- If fertile, willing to use effective birth control methods during the study
- Willing to undergo and able to tolerate frequent MRI or CT scan assessments during the study
- Able to understand and willing to complete symptom assessments using a patient-reported outcome instrument
- Provision of signed informed consent
Sites: There are currently seven research sites in the UK open
Glasgow – West of Scotland Beatson Centre
Gloucestershire Royal Hospital
Bart’s Hospital London
Guy’s Hospital London
Imperial College: Hammersmith Hospital, London
Churchill Hospital, Oxford
Royal Hallamshire Hospitals, Sheffield
Other: Clinical teams at participating hospitals will invite patients they deem eligible but please feel free to discuss with them.
Name: MYF3001: IMPACTMF
Title: A 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
Summary: An open label, randomized (2:1), Phase 3 study of imetelstat compared with best available therapy (BAT) in patients with intermediate 2 or high-risk MF refractory to JAKi treatment.
Patients will be randomized to receive imetelstat 9.4 mg/kg IV every three weeks or investigator selected BAT.
Eligible patients will be stratified based on
- a) Intermediate 2 or high-risk per Dynamic International Prognostic Scoring System;
- b) platelet count at entry (platelets ≥ 75 and < 150 x 109/L vs ≥ 150 x 109/L).
UK Chief Investigator: Professor Claire Harrison
Main Eligibility Main inclusion criteria
You may be able to join this study if all of the following apply.
You:
- are at least 18 years of age.
- Have a diagnosis of primary MF (PMF) or PET-MF or PPV-MF
- Have Dynamic International Prognostic Scoring System intermediate-2 or high-risk MF.
Are Refractory to JAK-inhibitor treatment after at least 6 months of treatment
Sites: There are currently four research sites in the UK open
Maidstone Hospital
St Bartholomew’s Hospital
Guys and St Thomas’ Hospital
Oxford University Hospitals
Other: Clinical teams at participating hospitals will invite patients they deem eligible but please feel free to discuss with them.
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.
