Comparing stem cell transplant and non-transplant therapies for high-risk myelofibrosis
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)
This study is trying to see if a stem cell transplant works better than other treatments for people with high-risk myelofibrosis.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 90 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | University Health Network, Toronto Academic / other |
| Drugs / interventions | ruxolitinib |
| Locations | 5 sites (Calgary, Alberta and 4 other locations) |
| Trial ID | NCT04217356 on ClinicalTrials.gov |
What this trial studies
This observational study aims to evaluate the effectiveness of hematopoietic stem cell transplantation (HCT) versus best available non-transplant therapies (BAT) in patients diagnosed with high-risk myelofibrosis. Participants will choose their preferred treatment option, either HCT or BAT, and their outcomes will be compared over time. The study seeks to provide insights into the risks and benefits of these treatment approaches, given the limited information currently available regarding optimal therapies for this condition.
Who should consider this trial
Good fit: Ideal candidates include individuals with a documented diagnosis of high-risk myelofibrosis and who meet specific eligibility criteria.
Not a fit: Patients who do not have high-risk myelofibrosis or those who are not eligible for either treatment option may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could help identify the most effective treatment options for patients with high-risk myelofibrosis, potentially improving patient outcomes.
How similar studies have performed: While there is limited information on similar studies, the comparison of HCT and non-transplant therapies in myelofibrosis is a relatively novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Calgary, Alberta and 4 other locations
- Tom Baker Cancer Centre — Calgary, Alberta, Canada (Recruiting)
- Cross Cancer Institute — Edmonton, Alberta, Canada (Not_yet_recruiting)
- St. Paul's Hospital — Vancouver, British Columbia, Canada (Not_yet_recruiting)
- Nova Scotia Health Authority — Halifax, Nova Scotia, Canada (Not_yet_recruiting)
- Princess Margaret Cancer Centre — Toronto, Ontario, Canada (Recruiting)
Study contacts
- Principal investigator: Vikas Gupta, M.D. — Princess Margaret Cancer Centre
- Study coordinator: Vikas Gupta, M.D.
- Email: vikas.gupta@uhn.ca
- Phone: 416-946-2885
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.