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

NCT04217356 | Active Not Recruiting

• 2 other identifiers: 19-6362ALLO-BAT
• Study Type: observational
• Target: 90
• Locations: 1 country
• Sites: 5

Brief 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.

Conditions

Bone Marrow Cancer; Myelofibrosis; High-Risk Cancer

Keywords

High-Risk Myelofibrosis; Stem Cell; Transplantation; Myelofibrosis; Bone marrow cancer

Outcome Measures

Primary Outcomes (4)

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

  5 years

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

  5 years

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

  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 from study allocation to death or last follow up.

  5 years

Secondary Outcomes (7)

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

  0 and 36 months

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

  0 and 36 months

• Median change in FACT-BMT Questionnaire

  0 and 36 months

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

  5 years

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

  5 years

Study Arms (2)

Hematopoietic stem cell transplant (HCT)

Standard of care hematopoietic stem cell transplant with a matched donor.

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.

Drug: Ruxolitinib; Drug: Hydroxyurea

Interventions

Hematopoietic stem cell transplant BIOLOGICAL

Intravenous infusion of hematopoietic stem cells from a donor.

Hematopoietic stem cell transplant (HCT)

Ruxolitinib DRUG

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

Also known as: JAKAVI

Best available non-transplant therapies (BAT)

Hydroxyurea DRUG

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

Best available non-transplant therapies (BAT)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Health Network, Toronto: lead

Study Sites (5)

Tom Baker Cancer Centre

Calgary, Alberta, T2N4N2, Canada

Location

Cross Cancer Institute

Edmonton, Alberta, T6G2G3, Canada

Location

St. Paul's Hospital

Vancouver, British Columbia, V6E1M7, Canada

Location

Nova Scotia Health Authority

Halifax, Nova Scotia, B3H2Y9, Canada

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G2M9, Canada

Location

Biospecimen

Retention: SAMPLES WITH DNA

Blood samples for future research related to hematological (blood) cancers and their treatments.

MeSH Terms

Conditions

Primary Myelofibrosis; Bone Marrow Neoplasms

Interventions

Stem Cell Transplantation; ruxolitinib; Hydroxyurea

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Hematologic Neoplasms; Neoplasms by Site; Neoplasms

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Urea; Amides; Organic Chemicals

Study Officials

• Vikas Gupta, M.D.

  Princess Margaret Cancer Centre

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 2, 2020
• First Posted: January 3, 2020
• Study Start: August 5, 2020
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: January 26, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (5)