Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: Trade Data, Destination Markets, and Economic Impact Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: Trade Data, Destination Markets, and Economic Impact
Clinical Trials in Canada / NCT04217356
Active, not recruiting Observational

Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis

NCT04217356 · tracked via the Priya Life Science Canada tracker
Phase
Observational
Started
2020-08-05
Last updated
2026-07-06

Condition(s) studied

MyelofibrosisHigh-Risk CancerBone Marrow Cancer

Investigational drug(s) / intervention(s)

Hematopoietic stem cell transplantRuxolitinib →Hydroxyurea →

Hematopoietic stem cell transplant: Intravenous infusion of hematopoietic stem cells from a donor.

Ruxolitinib: Ruxolitinib is type of drug called a janus kinase (JAK) inhibitor. Ruxolitinib is taken orally (by mouth).

Hydroxyurea: Hydroxyurea is a type of drug called an antimetabolite. Hydroxyurea is taken orally (by mouth).

Study summary

The purpose of this research study is to see how effective hematopoietic stem cell transplantation (HCT) is compared to best available non-transplant therapies (BAT) in patients with high risk myelofibrosis. This will be done by asking participants to choose the treatment that they prefer to receive (HCT or BAT) and then comparing the outcomes of the participants in both treatment groups.

Eligibility

Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
Inclusion Criteria: Recruitment Part: * Documented diagnosis of pre-fibrotic primary myelofibrosis (pre-fibrotic PMF), overt PMF, post-polycythemia MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF) confirmed by bone marrow biopsy * Have been tested or have results available for phenotypic driver mutations (JAK2/CALR/MPL) and high molecular risk (HMR) mutations using a broad myeloid malignancies targeted gene panel. * Eastern Cooperative Oncology Group (ECOG) performance status 0-2 * Able to provide informed consent * Adequate organ function * Donor search initiated or patient is agreeable to donor search * Meet the definition/criteria for high-risk myelofibrosis Study Arm Allocation: * Grade of fibrosis on bone marrow biopsy available according to World Health Organization (WHO) criteria * Results available for phenotypic driver mutations (JAK2/CALR/MPL) and targeted sequencing results using a broad myeloid malignancy panel with a minimal requirement to include results on High molecular risk (HMR) mutations such as ASXL1/EZH2/IDH1/IDH2/SRSF2/U2AF1/TP53 * ECOG performance status 0-2 * Adequate organ function * Information on donor search and donor type available Exclusion Criteria: Recruitment Part: * Blasts in peripheral blood or bone marrow ≥10% * For patients already on ruxolitinib at study entry, and meet the criteria of ruxolitinib failure * Previous history of transformation to blast phase or acute myeloid leukemia * Received allogeneic stem cell transplant for myeloproliferative neoplasm * Presence of an active uncontrolled infection * Myocardial infarction in the preceding 3 months * Active hepatitis A, B or C * Known human immunodeficiency virus (HIV) positive * History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer * Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation. * Pregnant or breastfeeding women Study Arm Allocation: * Blasts in peripheral blood or bone marrow ≥10% * Meet the criteria of ruxolitinib failure * Presence of an active uncontrolled infection * Myocardial infarction in the preceding 3 months * Active hepatitis A, B or C * Known HIV positive * History of active malignancy in the previous 2 years, except basal cell carcinoma or squamous cell carcinoma of skin or stage 0 cervical cancer * Pregnant or breastfeeding women * Any psychiatric illness or social circumstances or significant co-morbid conditions that will prevent patient from proceeding to allogeneic hematopoietic cell transplantation. * Time between registration and allocation of study arm \>24 weeks

Primary outcome measure(s)

Trial sites (5)

FacilityCityRegionStatus
Tom Baker Cancer Centre Calgary Alberta
Cross Cancer Institute Edmonton Alberta
St. Paul's Hospital Vancouver British Columbia
Nova Scotia Health Authority Halifax Nova Scotia
Princess Margaret Cancer Centre Toronto Ontario

On this site

📄 Jakafi (ruxolitinib) drug profile →

More University Health Network, Toronto trials in Canada

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04217356 on ClinicalTrials.gov ↗ ← All trials in Canada