Patients choose: transplant or Non-Transplant for rare bone marrow cancer?
NCT ID NCT04217356
First seen Jun 27, 2026 · Last updated Jul 07, 2026 · Updated 2 times
Summary
This study looks at two treatment options for people with a high-risk form of myelofibrosis, a rare bone marrow cancer. Participants choose whether they want a stem cell transplant or the best non-transplant treatments available. Researchers then track survival and quality of life to see which approach works better.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 90 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2020
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Patients with high-risk myelofibrosis including pre-fibrotic primary myelofibrosis (pre-fibrotic primary myelofibrosis), overt primary myelofibrosis, post-polycythemia myelofibrosis or post-essential thrombocythemia myelofibrosis.
- Ages
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18 to 70 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Cross Cancer Institute
Edmonton, Alberta, T6G2G3, Canada
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Nova Scotia Health Authority
Halifax, Nova Scotia, B3H2Y9, Canada
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Princess Margaret Cancer Centre
Toronto, Ontario, M5G2M9, Canada
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St. Paul's Hospital
Vancouver, British Columbia, V6E1M7, Canada
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Tom Baker Cancer Centre
Calgary, Alberta, T2N4N2, Canada
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as cure for myelofibrosis
- Can blood tests predict transplant complications?
- Can an immunotherapy combo boost remission in a rare lymphoma?
- Can diet and vitamin c supercharge rectal cancer treatment?
- Can a new daily pill shrink the spleen and ease myelofibrosis symptoms?
- Can a new pill tame myelofibrosis?