Ruxolitinib Pre-, During- and Post-HSCT for Patients With Primary or Secondary Myelofibrosis.

NCT03427866 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: 17-569
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 5

Brief Summary

This research study is studying a drug called Ruxolitinib as a possible treatment for Myelofibrosis.

Conditions

Myelofibrosis

Keywords

Myelofibrosis

Outcome Measures

Primary Outcomes (1)

• GVHD free and relapse free survival at 1 year

  The number of participants surviving after one year that have not experienced graft-versus host disease (GVHD) or relapse (GRFS rate)

  1 year

Secondary Outcomes (7)

• Progression Free Survival

  1 and 2 years

• Overall Survival

  1 and 2 years

• Cumulative incidence of aGVHD

  6 months

• Cumulative incidence of cGVHD

  1 and 2 years

• Rate of Engraftment

  2 years

Study Arms (2)

Ruxolitinib Eligible pre-HSCT

EXPERIMENTAL

* Ruxolitinib will be taken orally at a fixed dose twice every day * Dosing will be continuous, with a new cycle scheduled to start every 28 days. * There will be no break in dosing between cycles * Ruxolitinib can be administered with or without food.

Drug: Ruxolitinib

Ruxolitinib Not Eligible pre-HSCT

EXPERIMENTAL

* Ruxolitinib will be taken orally at a fixed dose twice every day after transplant * Dosing will be continuous, with a new cycle scheduled to start every 28 days. * There will be no break in dosing between cycles * Ruxolitinib can be administered with or without food.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Ruxolitinib is a medication that blocks certain proteins called tyrosine kinases. Specifically, it blocks tyrosine kinases called JAK2. The JAK2 pathway is over active in the disease, acute myeloid leukemia.

Also known as: Jakafi

Ruxolitinib Eligible pre-HSCT; Ruxolitinib Not Eligible pre-HSCT

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must have pathologically confirmed primary myelofibrosis according to WHO criteria1 or secondary myelofibrosis as defined by the IWG-MRT criteria.19
• Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria2 (Appendix 1) OR
• Intermediate-1 risk disease with one of the following additional unfavorable features known to impact the survival adversely
• Red cell transfusion dependency2
• Unfavorable Karyotype2
• Platelet count ≤100 x 109/L
• Age 18-75
• Participants must be designated to undergo reduced intensity allogeneic peripheral blood (PB) or bone marrow (BM) hematopoietic stem cell transplantation. Consent will be obtained prior to admission for HCT.
• Participants who will undergo HCT from the following donor types are eligible:
• /6 or 6/6 (HLA-A, B, DR) matched related donor
• /8 or 8/8 (HLA-A, B, DR, C) matched unrelated donor. Matching in the unrelated setting must be at the allele level
• ECOG performance status ≤2 (Karnofsky ≥60%, see Appendix A)
• Life expectancy of greater than 3 months
• Able to give informed consent
• Off all MF-directed therapy at the time of enrollment, with the exception of ruxolitinib

You may not qualify if:

• Hypersensitivity to any JAK inhibitor
• Prior allogeneic transplant for any hematopoietic disorder
• Had accelerated phase or leukemic transformation (≥10% blasts in PB or BM any time prior to HCT)
• Active uncontrolled infection
• History of another malignancy within 5-years of date of except h/o basal cell or squamous cell carcinoma of skin or Polycythemia Vera or Essential Thrombocythemia
• Patients without normal organ function defined as follows:
• AST (SGOT), ALT (SGPT) and Alkaline Phosphatase ≥ 3 × institutional Upper Limit of Normal (ULN)
• Direct bilirubin \>2.0 mg/dL
• Adequate renal function as defined by calculated creatinine clearance≤60 mL/min (Cockcroft-Gault formula)
• Have a chronic or active infection that requires systemic antibiotics, antifungal or antiviral treatment
• Have current or a history of congestive heart failure New York Heart Association (NYHA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF \< 40%, as measured by MUGA scan or echocardiogram)
• Pregnancy at the time of enrollment
• Unable to give informed consent
• Have an uncontrolled intercurrent illness including, but not limited to, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
• Subjects who require therapy with a strong CYP3A4 inhibitor prior to enrollment to this study

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Incyte Corporation: collaborator

Study Sites (5)

Massachusetts General Hospital

Boston, Massachusetts, 02214, United States

Location

Washington University

St Louis, Missouri, 63130, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Vanderbilt University

Nashville, Tennessee, 37235, United States

Location

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Gabriela Hobbs, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 2, 2018
• First Posted: February 9, 2018
• Study Start: August 28, 2018
• Primary Completion: May 1, 2024
• Study Completion: May 19, 2025
• Last Updated: July 29, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)