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

NCT ID: NCT04217356

Last Updated: 2025-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

90 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-08-05

Study Completion Date

2026-12-31

Brief Summary

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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.

Detailed Description

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There is currently little information regarding which treatments are best for patients with myelofibrosis. On one hand, hematopoietic stem cell transplantation (HCT) is potentially curative treatment but is associated with significant risk of complications related to graft failure (the new donor cells does not grow properly after the transplant), side effects such as graft versus host disease (the patient's cells attack the new donor cells), and risk of infections. Non-transplant therapies such as ruxolitinib provide effective symptom control for few months to few years, but are not curative in nature. As such, this study will compare the effectiveness of HCT versus best available non-transplant therapies (BAT) in patients with high risk myelofibrosis.

This is an observational study, meaning that participants will be followed to assess the effects of their treatment, but no intervention (treatments) will be given as a part of this study.

Conditions

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Myelofibrosis High-Risk Cancer Bone Marrow Cancer

Keywords

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High-Risk Myelofibrosis Stem Cell Transplantation Myelofibrosis Bone marrow cancer

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Hematopoietic stem cell transplant (HCT)

Standard of care hematopoietic stem cell transplant with a matched donor.

Hematopoietic stem cell transplant

Intervention Type BIOLOGICAL

Intravenous infusion of hematopoietic stem cells from a donor.

Best available non-transplant therapies (BAT)

Standard of care treatment with a janus kinase (JAK) inhibitor drug called ruxolitinib or treatment with an antimetabolite drug called hydroxyurea.

Ruxolitinib

Intervention Type DRUG

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

Hydroxyurea

Intervention Type DRUG

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

Interventions

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Hematopoietic stem cell transplant

Intravenous infusion of hematopoietic stem cells from a donor.

Intervention Type BIOLOGICAL

Ruxolitinib

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

Intervention Type DRUG

Hydroxyurea

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

Intervention Type DRUG

Other Intervention Names

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JAKAVI

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Health Network, Toronto

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Vikas Gupta, M.D.

Role: PRINCIPAL_INVESTIGATOR

Princess Margaret Cancer Centre

Locations

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Tom Baker Cancer Centre

Calgary, Alberta, Canada

Site Status RECRUITING

Cross Cancer Institute

Edmonton, Alberta, Canada

Site Status NOT_YET_RECRUITING

St. Paul's Hospital

Vancouver, British Columbia, Canada

Site Status NOT_YET_RECRUITING

Nova Scotia Health Authority

Halifax, Nova Scotia, Canada

Site Status NOT_YET_RECRUITING

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

Site Status RECRUITING

Countries

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Canada

Central Contacts

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Vikas Gupta, M.D.

Role: CONTACT

Phone: 416-946-2885

Email: [email protected]

Facility Contacts

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Sonia Cerquozzi, M.D.

Role: primary

Elena Liew, M.D.

Role: primary

Lynda Foltz, M.D.

Role: primary

Mahmoud Elsawy, M.D.

Role: primary

Vikas Gupta, M.D.

Role: primary

Other Identifiers

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ALLO-BAT

Identifier Type: OTHER

Identifier Source: secondary_id

19-6362

Identifier Type: -

Identifier Source: org_study_id