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Clinical Trials in Canada

Initiated by the discovery of the JAK2 mutation, the past decade has seen a great deal of activity in the development of potential new therapies for MPNs. Several JAK inhibitor drugs are in clinical trials as well as other therapy types such as stem cell transplant.

Should I participate in a clinical trial?

The decision to take part in a clinical trial is a personal one and should be made with the input of your physician. Clinical trials can provide access to new medications that may be of benefit for treatment of your disease, and the data from clinical trials is of great benefit in helping future patients affected with similar condition. But you also need to consider that these medications are usually experimental and some of the adverse effects may not be known. Participating in a clinical trial may mean a time commitment, with more frequent visits to the trial centre.

Trials for Myelofibrosis

Study of imetelstat in JAK-treated patients with myelofibrosis

Study to evaluate activity of 2 dose levels of imetelstat in participants with intermediate-2 or high-risk myelofibrosis (MF) previously treated with Janus kinase (JAK) inhibitor

This study is to evaluate the spleen response and symptom response of 2 different doses of imetelstat in participants with intermediate-2 or high-risk myelofibrosis (MF) who are relapsed after or are refractory to Janus kinase (JAK) inhibitor treatment

Who can participate?

Inclusion criteria
  • Diagnosis of MF
  • Enlarged spleen
  • Refractory or relapsed after JAK inhibitor therapy
Exclusion criteria
  • Prior treatment with imetelstat
  • Prior stem cell transplant

Where are trials being held?

Jewish General Hospital, Montreal

Study of the safety of PIM447 in combination with ruxolitinib (INC424) and LEE011 in patients with myelofibrosis

A phase Ib, multi-center, open-label, dose-escalation study of PIM447 in combination with ruxolitinib (INC424) and LEE011 administered orally in patients with myelofibrosis

This study will investigate the safety of the triple drug combination of (1) PIM447 + ruxolitinib + LEE011, (2) ruxolitinib + PIM447 and (3) ruxolitinib + LEE011 in treating myelofibrosis patients.

Who can participate?

Inclusion criteria include
  • Diagnosis of JAKV617F positive primary MF
  • Previous treatment with ruxolitinib
  • AND had poor response to ruxolitinib
Exclusion criteria include
  • Intolerance to ruxolitinib
  • Prior treatment with CDK 4/6 inhibitor or PIM inhibitor

Where are trials being held?

Screening for asymptomatic portal vein thrombosis and portal hypertension in patients with MPN

Portal vein thrombosis and portal hypertension in MPNs

Portal vein thrombosis and portal hypertension are serious complications of myeloproliferative neoplasms (MPNs) but are often not diagnosed until a late stage when patients are symptomatic.  This study is screening for portal vein thrombosis and portal hypertension in patients with Philadelphia negative MPNs – MF, ET and PV.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF, ET or PV.
Exclusion criteria include
  • History of portal vein thrombosis, Budd-chiari syndrome, esophageal varices or cirrhosis.

Where are trials being held?

A study to evaluate efficacy and safety of vismodegib (Erivedge) in combination with ruxolitinib for the treatment of intermediate- or high-risk myelofibrosis

A Phase Ib/III, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Vismodegib in Combination with Ruxolitinib versus Placebo and Ruxolitinib in Patients with Intermediate or High risk Myelofibrosis (MYLIE)

This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of vismodegib plus (+) ruxolitinib versus placebo + ruxolitinib in participants with intermediate- or high-risk MF.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF
  • Intermediate-1, intermediate-2 or high risk disease
  • Splenomegaly
  • Adequate hepatic and renal function
Exclusion criteria include
  • Prior treatment with Hedgehog or JAK inhibitor
  • Inadequate bone marrow function
  • Recent (within 28 days) treatment with certain drugs

Where are trials being held?

A phase 2 study of PRM-151 in patients with myelofibrosis

A phase 2, open-label, prospective study of PRM-151 in subjects with primary myelofibrosis (PMF), post-polycythemia vera MF (post-PV MF), or post-essential thrombocythemia MF (post-ET MF)

The purpose of this phase 2 study is to gather information on whether PRM-151 has an effect on the MF disease, whether it is safe in patients with MF, and how well it is tolerated.

Who can participate?

Inclusion criteria include
  • Diagnosis of intermediate-1, intermediate-2 or high-risk MF
  • Not a candidate for ruxolitinib
Exclusion criteria include
  • High white blood cell count
  • Peripheral blood blasts greater than 10%

Where are trials being held?

SL-401 in advanced, high risk MPNs

SL-401 in patients with advanced, high risk myeloproliferative neoplasms (MPNs)

This is a phase1/2  non-randomized open label multi-center study to determine the safety of the drug SL-401 in patients with high-risk myeloproliferative neoplasms (systemic mastocytosis [SM], advanced symptomatic hypereosinoophic disorder [PED], myelofibrosis [MF], and chronic myelomonocytic leukemia [CMML]).

Who can participate?

Inclusion criteria include
  • Adequate baseline organ function (including cardiac, renal and hepatic)
  • Must exceed minimum set absolute neutrophil count
Exclusion criteria include
  • Prior treatment with SL-401
  • Hypersensitivity to any drug components
  • Any medical conditions that put the participant at unacceptably high risk of toxicities

Where are trials being held?

Momelotinib in transfusion-dependent adults with primary myelofibrosis or post-polycythemia vera or post-essential thrombocythemia myelofibrosis (not recruiting)

A Phase 2 open label translational biology study of momelotinib in transfusion dependent subjects with primary myelofibrosis (PMF) or post polycythemia vera or post essential thrombocythemia myelofibrosis (PPV-MF or PET-MF)

Note: this trial is not recruiting participants

This phase 2 study will evaluate the rate at which transfusion-dependent adults with myelofibrosis become transfusion indepdnent after treatment with momelotinib.

Who can participate?

Inclusion criteria include
  • Diagnosis of intermediate-2 or high-risk MF
  • Transfusion dependent at baseline
Exclusion criteria include
  • Splenectomy
  • Prior treatment with momelotinib
  • Treatment with a JAK inhibitor within 21 days before commencing trial

Where are trials being held?

Efficacy and safety of ruxolitinib in early myelofibrosis patients with high molecular risk mutations (terminated)

Note: this trial has been terminated

A Randomized, Double Blind, Placebo-controlled, Multicenter, Phase III Study Investigating the Efficacy and Safety of Ruxolitinib in Early Myelofibrosis Patients With High Molecular Risk Mutations

This phase 3 study aims to determine whether myelofibrosis patients who have high risk mutations benefit from receiving ruxolitinib treatment earlier in their disease.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF
  • Bone marrow fibrosis at least grade 1
  • At least one mutation in one of the five high molecular risk (HMR) genes (ASXL1, EZH2, SRSF2 and IDH1/2)
  • Splenomegaly
Exclusion criteria include
  • Previous treatment with ruxolitinib or any other JAK1/2 inhibitor

Where are trials being held?

Momelotinib versus ruxolitinib in patients with myelofibrosis (not recruiting)

Note: this trial is not recruiting participants

A phase 3, randomized, double-blind, active-controlled study evaluating momelotinib vs. ruxolitinib in subjects with primary myelofibrosis (PMF) or post-polycythemia vera or post-essential thrombocythemia myelofibrosis (Post-PV/ET MF)

The purpose of this study is to investigate the efficacy of momelotinib versus ruxolitinib in MF patients who have not previously been treated with a Janus kinase (JAK) inhibitor.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF
  • Symptomatic splenomegaly, hepatomegaly, anemia
  • AND/OR unresponsive to available therapy
Exclusion criteria include
  • Splenectomy
  • Uncontrolled intercurrent illness
  • Prior use of JAK1 or JAK2 inhibitor

Where are trials being held?

Oral pacritinib versus best available therapy to treat myelofibrosis with thrombocytopenia - (terminated)

Note: this trial has been terminated

A randomized controlled phase 3 study of oral pacritinib versus best available therapy in patients with thrombocytopenia and primary myelofibrosis, post polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis-PAC326

This study will investigate the hypothesis that treatment with pacritinib results in a greater reduction in spleen size and symptoms in patients with MF and low platelet count (thrombocytopenia) compared with the best available therapy.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF
  • Low platelet count
  • At least 2 weeks since receiving treatment for myelofibrosis
Exclusion criteria include
  • Prior treatment with more than 2 JAK inhibitors or pacritinib
  • Uncontrolled intercurrent illness
  • Clinically symptomatic and uncontrolled cardiovascular disease

Where are trials being held?

Momelotinib versus best available therapy in myelofibrosis patients with anemia or thrombocytopenia (not recruiting)

Note: this trial is not recruiting participants

A phase 3, randomized study to evaluate the efficacy of momelotinib versus best available therapy in anemic or thrombocytopenic subjects with primary myelofibrosis, post-polycythema vera myelofibrosis, or post-essential thrombocythemia myelofibrosis who were treated with ruxolitinib

This study will investigate the efficacy of momelotinib versus best available therapy for MF patients with anemia or low platelet count (thrombocytopenia) who were treated with ruxolitinib.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF
  • Currently or previously treated with ruxolitinib
  • AND had anemia or low platelet count
Exclusion criteria include
  • Splenectomy
  • Prior treatment with momelotinib
  • Uncontrolled intercurrent illness

Where are trials being held?

Ruxolitinib in MF patients prior to hematopoietic cell transplantation (not recruiting)

Exploring the potential of dual kinase JAK 1/2 inhibitor ruxolitinib (INC424) with reduced intensity allogeneic hematopoietic cell transplantation in patients with myelofibrosis

Note: this trial is not recruiting participants

Ruxolitinib is approved for treatment of patients with MF but its use prior to hematopoietic cell transplant (bone marrow transplant) is experimental.  This study will investigate whether treatment with ruxolitinib prior to transplant in patients with advanced MF will be successful and beneficial.

Who can participate?

Inclusion criteria include
  • Age 18-70 years
  • Diagnosis of MF
  • Availability of matched donor
Exclusion criteria include
  • Prior treatment with a JAK inhibitor other than ruxolitinib
  • Cirrhosis
  • Prior allogeneic transplant for any hematopoietic disorder

Where are trials being held?

Trials for Polycythemia Vera

Screening for asymptomatic portal vein thrombosis and portal hypertension in patients with MPN

Portal vein thrombosis and portal hypertension in MPNs

Portal vein thrombosis and portal hypertension are serious complications of myeloproliferative neoplasms (MPNs) but are often not diagnosed until a late stage when patients are symptomatic.  This study is screening for portal vein thrombosis and portal hypertension in patients with Philadelphia negative MPNs – MF, ET and PV.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF, ET or PV.
Exclusion criteria include
  • History of portal vein thrombosis, Budd-chiari syndrome, esophageal varices or cirrhosis.

Where are trials being held?

Princess Margaret Cancer Centre, Toronto

Ruxolitinib in patients with polycythemia vera for whom hydroxyurea treatment failed

Expanded treatment protocol (ETP) of ruxolitinib in patients with polycythemia vera who are hydroxyurea resistant or intolerant and for whom no treatment alternatives are available

This study is to provide early access to ruxolitnib in PV patients who are resistant or intolerant to hydroxyurea and for whom no other treatment option exists. The study will provide safety information on ruxolitinib in these patients.

Who can participate?

Inclusion criteria
  • Diagnosis of PV
  • Resistant or intolerant to hydroxyurea
  • No access to a comparable or satisfactory alternative treatment
Exclusion criteria
  • None provided

Where are trials being held?

St Paul’s Hospital, Vancouver (Providence Hematology)

Trials for Essential Thrombocythemia

Screening for asymptomatic portal vein thrombosis and portal hypertension in patients with MPN

Portal vein thrombosis and portal hypertension in MPNs

Portal vein thrombosis and portal hypertension are serious complications of myeloproliferative neoplasms (MPNs) but are often not diagnosed until a late stage when patients are symptomatic.  This study is screening for portal vein thrombosis and portal hypertension in patients with Philadelphia negative MPNs – MF, ET and PV.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF, ET or PV.
Exclusion criteria include
  • History of portal vein thrombosis, Budd-chiari syndrome, esophageal varices or cirrhosis.

Where are trials being held?

Princess Margaret Cancer Centre, Toronto

Related to this topic...

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