Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis (ALLO-BAT)

January 22, 2026 updated by: University Health Network, Toronto

A Patient Preferences-Controlled Study of Allogeneic Hematopoietic Cell Transplantation Versus Best Available Non-Transplant Therapies in Patients With High-Risk Myelofibrosis (ALLO-BAT Study)

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Observational

Enrollment (Estimated)

90

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N4N2
        • Tom Baker Cancer Centre
      • Edmonton, Alberta, Canada, T6G2G3
        • Cross Cancer Institute
    • British Columbia
      • Vancouver, British Columbia, Canada, V6E1M7
        • St. Paul's Hospital
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H2Y9
        • Nova Scotia Health Authority
    • Ontario
      • Toronto, Ontario, Canada, M5G2M9
        • Princess Margaret Cancer Centre

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Hematopoietic stem cell transplant (HCT)
Standard of care hematopoietic stem cell transplant with a matched donor.
Biological: Hematopoietic stem cell transplant
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.
Drug: Ruxolitinib
Ruxolitinib is type of drug called a janus kinase (JAK) inhibitor. Ruxolitinib is taken orally (by mouth).
Other Names:
  • JAKAVI
Drug: Hydroxyurea
Hydroxyurea is a type of drug called an antimetabolite. Hydroxyurea is taken orally (by mouth).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of patients allocated to hematopoietic stem cell transplantation (HCT)
Time Frame: 5 years
5 years
Number of patients allocated to best available non-transplant therapies (BAT)
Time Frame: 5 years
5 years
Overall survival rate of patients who receive hematopoietic stem cell transplantation (HCT)
Time Frame: 5 years
Time from study allocation to death or last follow up.
5 years
Overall survival rate of patients who receive best available non-transplant therapies (BAT)
Time Frame: 5 years
Time from study allocation to death or last follow up.
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Median change in Patient Global Impression of Change (PGIC) score
Time Frame: 0 and 36 months
Range from -3 to 3. Positive number equals increase in quality of life.
0 and 36 months
Median change in MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
Time Frame: 0 and 36 months
Range from 0 to 10. Increase equals worsening of symptoms.
0 and 36 months
Median change in FACT-BMT Questionnaire
Time Frame: 0 and 36 months
Range from 1 to 4. Increase equals increase in quality of life.
0 and 36 months
Disease-free survival of patients who receive hematopoietic stem cell transplantation (HCT)
Time Frame: 5 years
Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.
5 years
Disease-free survival of patients who receive best available non-transplant therapies (BAT)
Time Frame: 5 years
Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.
5 years
Number of patients who receive hematopoietic stem cell transplantation (HCT) in remission (complete and partial)
Time Frame: 3 years
3 years
Number of patients who receive best available non-transplant therapies (BAT) in remission (complete and partial)
Time Frame: 3 years
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Health Network, Toronto

Investigators

  • Principal Investigator: Vikas Gupta, M.D., Princess Margaret Cancer Centre

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 5, 2020

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

December 31, 2026

Study Registration Dates

First Submitted

January 2, 2020

First Submitted That Met QC Criteria

January 2, 2020

First Posted (Actual)

January 3, 2020

Study Record Updates

Last Update Posted (Actual)

January 26, 2026

Last Update Submitted That Met QC Criteria

January 22, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 19-6362
  • ALLO-BAT (Other Identifier: Princess Margaret Cancer Centre)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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