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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 CPI-610 with and without ruxolitinib in patients with myelofibrosis

A phase 2 study of CPI-610 with and without ruxolitinib in patients with myelofibrosis

This study is to evaluate the efficacy and safety of CPI-610 in patients with myelofibrosis.  There are three treatment groups (arms): (1) CPI-610 monotherapy, (2) combination therapy of CPI-610 and ruxolitinib, and (3) CPI-610 and ruxolitinib combination therapy in patients not doing well on ruxolitinib therapy or are ruxolitinib naive.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF
  • Enlarged spleen
  • Other criteria depend on the treatment group (arm)
Exclusion criteria include
  • Prior treatment with a BET inhibitor
  • Impaired cardiac function

Where are trials being held?

Jewish General Hospital, Montreal

Princess Margaret Cancer Centre, Toronto

Juravinski Cancer Centre, Hamilton

St Paul’s Hospital (Providence Hematology), Vancouver

University of Alberta, Edmonton

Study of fedratinib in patients with myelofibrosis previously treated with ruxolitinib

An efficacy and safety trial of fedratinib in subjects with DIPSS, intermediate or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-esential thrombocythemia myelofibrosis and previously treated with ruxolitinib (FREEDOM)

This study is to evaluate the safety and efficacy of fedratinib in patients with intermediate/high-risk myelofibrosis who have been previously treated with ruxolitinib.

Who can participate?

Inclusion criteria include
  • Diagnosis of MF
  • DIPSS risk score of intermediate or high
  • Enlarged spleen
  • Previously exposed to ruxoitinib
Exclusion criteria include
  • Splenectomy
  • Prior stem cell transplant

Where are trials being held?

Princess Margaret Cancer Centre, Toronto

Study of imetelstat in JAK-treated patients with myelofibrosis (not recruiting)

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

Note: this trial is not recruiting participants

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

Where are trials being held?

Jewish General Hospital, Montreal

Screening for asymptomatic portal vein thrombosis and portal hypertension in patients with MPN (accrual completed)

Portal vein thrombosis and portal hypertension in MPNs

Note: this trial is not recruiting participants

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 phase 2 study of PRM-151 in patients with myelofibrosis (accrual completed)

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) 

Note: this trial is not recruiting participants

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?

Trials for Polycythemia Vera & Essential Thrombocythemia

Study of idasanutlin monotherapy in participants with hydroxyurea-resistant/intolerant PV

Sudy to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Idasanutlin Monotherapy in Participants with Hydroxyurea-Resistant/Intolerant Polycythemia Vera

This study is to evaluate efficacy and safety of idasanutlin therapy in patients with polycythemia vera (PV) who are resistant or intolerant to hydroxyurea therapy.

Who can participate?

Inclusion criteria include
  • Diagnosis of polycythemia vera (PV)
  • Hematocrit at initiation of idasanutlin > 40%
  • Phlebotomy dependent
  • Resistance to/intolerance to hydroxyurea
Exclusion criteria include
  • Diagnosis of post-polycythemia myelofibrosis (PPV-MF
  • Clinically-significant thrombosis within 3 months of screening
  • Received certain therapies (see ‘Learn More’ for details)

Where are trials being held?

Princess Margaret Cancer Centre, Toronto

Screening for asymptomatic portal vein thrombosis and portal hypertension in patients with MPN (accrual completed)

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

Trials for Mastocytosis

Study to evaluate the efficacy and safety of Avapritinib (BLU-285) in advanced systemic mastocytosis

An open-label, single arm, phase 2 study to evaluate efficacy and safety of avapritinib (BLU-285), a selective KIT mutation-targated tyrosine kinase inhibitor, in patients with advanced, systemic mastocytosis

This study is to evaluate the efficacy and safety of avapritinib in patients with systemic mastocytosis.  Systemic mastocytosis includes aggressive systemic mastocytosis, systemic mastocytosis with associatated hematologic neoplasm, and mast cell leukemia.

Who can participate?

Inclusion criteria include
  • Diagnosis of aggressive systemic mastocytosis, systemic mastocytosis with associatated hematologic neoplasm, or mast cell leukemia
  • At least one measureable C finding, per modified IWG-MRT-ECNM and evaluatble for response assessment (unless diagnosis is mast cell leukemia).
Exclusion criteria include
  • Prior treatment avapritnib
  • Any cytoreductive therapy within 14 days of screening bone marrow biopsy
  • Cladribine, INF-alpha, pegylated-INF or antibody therapy within 28 days of screening bone marrow biopsy
  • Radiotherapy within 14 days of screening bone marrow biopsy
  • Hematopoietic growth factor within 14 days of screening biopsy

Where are trials being held?

St Michael’s Hospital, Toronto

Related to this topic...

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