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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04217356
Other study ID # 19-6362
Secondary ID ALLO-BAT
Status Recruiting
Phase
First received
Last updated
Start date August 5, 2020
Est. completion date February 5, 2026

Study information

Verified date March 2024
Source University Health Network, Toronto
Contact Vikas Gupta, M.D.
Phone 416-946-2885
Email vikas.gupta@uhn.ca
Is FDA regulated No
Health authority
Study type Observational

Clinical 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. 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.


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.


Recruitment information / eligibility

Status Recruiting
Enrollment 90
Est. completion date February 5, 2026
Est. primary completion date August 5, 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years to 70 Years
Eligibility 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 Design


Intervention

Biological:
Hematopoietic stem cell transplant
Intravenous infusion of hematopoietic stem cells from a donor.
Drug:
Ruxolitinib
Ruxolitinib is type of drug called a janus kinase (JAK) inhibitor. Ruxolitinib is taken orally (by mouth).
Hydroxyurea
Hydroxyurea is a type of drug called an antimetabolite. Hydroxyurea is taken orally (by mouth).

Locations

Country Name City State
Canada Tom Baker Cancer Centre Calgary Alberta
Canada Cross Cancer Institute Edmonton Alberta
Canada Nova Scotia Health Authority Halifax Nova Scotia
Canada Princess Margaret Cancer Centre Toronto Ontario
Canada St. Paul's Hospital Vancouver British Columbia

Sponsors (1)

Lead Sponsor Collaborator
University Health Network, Toronto

Country where clinical trial is conducted

Canada, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of patients allocated to hematopoietic stem cell transplantation (HCT) 5 years
Primary Number of patients allocated to best available non-transplant therapies (BAT) 5 years
Primary Overall survival rate of patients who receive hematopoietic stem cell transplantation (HCT) Time from study allocation to death or last follow up. 5 years
Primary Overall survival rate of patients who receive best available non-transplant therapies (BAT) Time from study allocation to death or last follow up. 5 years
Secondary Median change in Patient Global Impression of Change (PGIC) score Range from -3 to 3. Positive number equals increase in quality of life. 0 and 36 months
Secondary Median change in MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) Range from 0 to 10. Increase equals worsening of symptoms. 0 and 36 months
Secondary Median change in FACT-BMT Questionnaire Range from 1 to 4. Increase equals increase in quality of life. 0 and 36 months
Secondary 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. 5 years
Secondary 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. 5 years
Secondary Number of patients who receive hematopoietic stem cell transplantation (HCT) in remission (complete and partial) 3 years
Secondary Number of patients who receive best available non-transplant therapies (BAT) in remission (complete and partial) 3 years
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