The newest options/drug trials

Trials offer access to unproven but potentially beneficial therapies.

JAK2 inhibitor drugs are under trial in several countries and are showing promise in improving quality of life for people with myelofibrosis. Other treatments are under trial for people with essential thrombocythaemia and polycythaemia vera – and we hope to find drugs that are more effective while causing fewer side effects.

If you are interested in enrolling in a trial and live in the U.K. please email us at info@mpdvoice.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 the JAK2 inhibitor 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.
  • NCI The National Cancer Institute (NCI)’s clinical trials page offers detailed information on trials taking place in the US – search by disease name, for instance “myelofibrosis”.

You can also watch a video interview of Albert, who is enrolled in a JAK2 inhibitor trial. You can read about people who are taking the newest drugs in Real Stories.

Trials open for myelofibrosis

  • Phase II study of SAR302503 in Myelofibrosis patients previously treated with Ruxolitinib (JAKARTA2) (trial recruiting in U.K., U.S.A., Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Spain)
  • Exploring the potential of Dual Kinase JAK 1/2 inhibitor Ruxolitinib (INC424) with reduced intensity allogeneic hematopoietic cell transplantation in patients with Myelofibrosis (trial recruiting in U.K., U.S.A., Canada, Israel, Italy)
  • An assessment of safety and efficacy of Ruxolitinib (INCB018424) in patients with primary Myelofibrosis (PMF), post Essential Thrombocythaemia-myelofibrosis (PPV-MF) and post Polycythaemia Vera-myelofibrosis (PET-MF) who have platelet counts of 50 x 10^9/l to 100 x 10^9/l (trial recruiting in U.S.A. only)
  • Phase III study of Pacritinib vs BAT (best available therapy) in patients with Myelofibrosis, Post PV-MF and Post ET-MF (trial recruiting in multiple international sites)
  • Phase Ib/II dose finding study to assess the safety and efficacy of LDE225 + INC424 (JAK inhibitor) in patients with Myelofibrosis (not yet open - U.K., Australia, Canada, Germany, Spain)
  • Panobinostat and Ruxolitinib in primary-Myelofibrosis, post-Polycythaemia Vera-Myelofibrosis or post-Essential Thrombocythaemia-myelofibrosis (trial recruiting in U.K., France, Germany, Ireland, Italy)
  • A study to find the maximum tolerated dose of the experimental combination of the drugs INC424 and BKM120 in patients with primary or secondary Myelofibrosis (trail recruiting in U.K., Australia, Austria, France, Germany, Israel, Italy, Singapore, Spain)

Trials open for polycythaemia vera

  • RESPONSE, a trial looking at the effects of Ruxolitinib (trial recruiting in U.K. only)
  • MAJIC, a clinical trial for people who have had problems or lack of response to Hydroxycarbamide (Hydrea) (trial recruiting in UK only)
  • MPDRC 112 a trial comparing Hydroxycarbamide (Hydrea) with Pegylated Interferon 2a (PEGASYS) in newly diagnosed high risk PV patients (trial recruiting in UK only)
  • Switch Study from Hydroxycarbamide (Hydrea) to Ruxolitinib for RELIEF of Polycythaemia Vera symptoms: The Relief Study (trial recruiting in U.K., U.S.A., Belgium, Ireland, Italy, Spain)

Trials open for essential thrombocythaemia

  • MAJIC, a clinical trial for people who have had problems or lack of response to Hydroxycarbamide (Hydrea) (trial recruiting in UK only)
  • PT-1 study for people with low risk ET (trial recruiting in UK only)MPDRC 112 a trial comparing Hydroxycarbamide (Hydrea) with Pegylated Interferon 2a (PEGASYS) in newly diagnosed high risk ET patients (trial recruiting in UK only)

Combined Trials for polycythaemia/essential thrombocythaemia

  • A randomized phase II study of the efficacy and safety of orally administered SAR302503 in patients with Polycythaemia Vera or Essential Thrombocythaemia who are resistant or intolerant to Hydroxycarbamide (Hydrea) (trial recruiting in U.K., U.S.A., Australia, Canada, France, Germany, Italy, Korea, Spain)
  • A randomized trial of Pegylated Interferon Alfa-2a versus Hydroxycarbamide (Hydrea) in Polycythaemia Vera and Essential Thrombocythaemia who are newly diagnosed (trial recruiting in U.K., U.S.A, Belgium, France, Ireland, Italy, Netherlands, Sweden)