- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04217356
Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis (ALLO-BAT)
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)
Study Overview
Status
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
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Vikas Gupta, M.D.
- Phone Number: 416-946-2885
- Email: vikas.gupta@uhn.ca
Study Locations
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Alberta
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Calgary, Alberta, Canada, T2N4N2
- Recruiting
- Tom Baker Cancer Centre
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Contact:
- Sonia Cerquozzi, M.D.
- Phone Number: 403-944-5948
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Principal Investigator:
- Sonia Cerquozzi, M.D.
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Edmonton, Alberta, Canada, T6G2G3
- Not yet recruiting
- Cross Cancer Institute
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Contact:
- Elena Liew, M.D.
- Phone Number: 780-417-1584
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Principal Investigator:
- Elena Liew, M.D.
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British Columbia
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Vancouver, British Columbia, Canada, V6E1M7
- Not yet recruiting
- St. Paul's Hospital
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Contact:
- Lynda Foltz, M.D.
- Phone Number: 64986 604-682-2344
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Principal Investigator:
- Lynda Foltz, M.D.
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Nova Scotia
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Halifax, Nova Scotia, Canada, B3H2Y9
- Not yet recruiting
- Nova Scotia Health Authority
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Contact:
- Mahmoud Elsawy, M.D.
- Phone Number: 902-473-7006
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Principal Investigator:
- Mahmoud Elsawy, M.D.
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Ontario
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Toronto, Ontario, Canada, M5G2M9
- Recruiting
- Princess Margaret Cancer Centre
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Contact:
- Vikas Gupta, M.D.
- Phone Number: 416-946-2885
-
Principal Investigator:
- Vikas Gupta, M.D.
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
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
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.
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Intravenous infusion of hematopoietic stem cells from a donor.
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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 is type of drug called a janus kinase (JAK) inhibitor.
Ruxolitinib is taken orally (by mouth).
Other Names:
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.
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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
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Range from -3 to 3. Positive number equals increase in quality of life.
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0 and 36 months
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Median change in MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
Time Frame: 0 and 36 months
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Range from 0 to 10. Increase equals worsening of symptoms.
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0 and 36 months
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Median change in FACT-BMT Questionnaire
Time Frame: 0 and 36 months
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Range from 1 to 4. Increase equals increase in quality of life.
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0 and 36 months
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Disease-free survival of patients who receive hematopoietic stem cell transplantation (HCT)
Time Frame: 5 years
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Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.
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5 years
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Disease-free survival of patients who receive best available non-transplant therapies (BAT)
Time Frame: 5 years
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Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.
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5 years
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Number of patients who receive hematopoietic stem cell transplantation (HCT) in remission (complete and partial)
Time Frame: 3 years
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3 years
|
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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
Investigators
- Principal Investigator: Vikas Gupta, M.D., Princess Margaret Cancer Centre
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Neoplasms
- Neoplasms by Site
- Bone Marrow Diseases
- Hematologic Diseases
- Myeloproliferative Disorders
- Hematologic Neoplasms
- Primary Myelofibrosis
- Bone Marrow Neoplasms
- Molecular Mechanisms of Pharmacological Action
- Nucleic Acid Synthesis Inhibitors
- Enzyme Inhibitors
- Antineoplastic Agents
- Antisickling Agents
- Hydroxyurea
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)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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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