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MyelofibrosisALLO-BATNCT04217356

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

An observational study of Myelofibrosis and High-Risk Cancer, run by University Health Network, Toronto at 5 sites in Canada.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
Hematopoietic stem cell transplant, Ruxolitinib and Hydroxyurea
How many people
90 participants
Where
5 sitesCanada
Study dates
5 Aug 2020to 31 Dec 2026
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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.

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

Canada5 sites

  • Tom Baker Cancer Centre

    Calgary, Alberta, T2N4N2

  • Cross Cancer Institute

    Edmonton, Alberta, T6G2G3

  • Nova Scotia Health Authority

    Halifax, Nova Scotia, B3H2Y9

  • Princess Margaret Cancer Centre

    Toronto, Ontario, M5G2M9

  • St. Paul's Hospital

    Vancouver, British Columbia, V6E1M7

Other trials in Canada

About this study

Official title: 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.

Read the 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.

Being tested
Hematopoietic stem cell transplant, Ruxolitinib, Hydroxyurea
Keywords
High-Risk Myelofibrosis, Stem Cell, Transplantation, Myelofibrosis, Bone marrow cancer
Medical subject headings
Primary Myelofibrosis, Bone Marrow Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Hematopoietic stem cell transplantBiological

    Intravenous infusion of hematopoietic stem cells from a donor.

  • RuxolitinibDrug

    Also called JAKAVI

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

  • HydroxyureaDrug

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

Study groups in NCT04217356
Study groupRoleWhat they receive
Hematopoietic stem cell transplant (HCT)Standard of care hematopoietic stem cell transplant with a matched donor.
Receives: Biological: Hematopoietic stem cell transplant
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.
Receives: Drug: Ruxolitinib; Drug: Hydroxyurea

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of patients allocated to hematopoietic stem cell transplantation (HCT)

    Measured: 5 years

  2. Number of patients allocated to best available non-transplant therapies (BAT)

    Measured: 5 years

  3. Overall survival rate of patients who receive hematopoietic stem cell transplantation (HCT)

    Time from study allocation to death or last follow up.

    Measured: 5 years

  4. Overall survival rate of patients who receive best available non-transplant therapies (BAT)

    Time from study allocation to death or last follow up.

    Measured: 5 years

Secondary outcomes (7)

  1. Median change in Patient Global Impression of Change (PGIC) score

    Range from -3 to 3. Positive number equals increase in quality of life.

    Measured: 0 and 36 months

  2. Median change in MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

    Range from 0 to 10. Increase equals worsening of symptoms.

    Measured: 0 and 36 months

  3. Median change in FACT-BMT Questionnaire

    Range from 1 to 4. Increase equals increase in quality of life.

    Measured: 0 and 36 months

  4. Disease-free survival of patients who receive hematopoietic stem cell transplantation (HCT)

    Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.

    Measured: 5 years

  5. Disease-free survival of patients who receive best available non-transplant therapies (BAT)

    Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.

    Measured: 5 years

  6. Number of patients who receive hematopoietic stem cell transplantation (HCT) in remission (complete and partial)

    Measured: 3 years

  7. Number of patients who receive best available non-transplant therapies (BAT) in remission (complete and partial)

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Jan 2020
  2. Study start5 Aug 2020
  3. Primary completion31 Dec 2026 (estimated)
  4. Study completion31 Dec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
90 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04217356
Sponsor's protocol ID
19-6362
Other IDs
ALLO-BAT
Acronym
ALLO-BAT
Status last verified
Jul 2026
First submitted
2 Jan 2020
Last updated
6 Jul 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

An observational study of Myelofibrosis and High-Risk Cancer, run by University Health Network, Toronto at 5 sites in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 6 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of patients allocated to hematopoietic stem cell transplantation (HCT) (time frame: 5 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04217356.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2026.

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