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

NCT03427866 | Completed Phase 2 Results 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 year and 2 year

• Cumulative Incidence of aGVHD

  6 months

• Cumulative Incidence of cGVHD

  2 years

• Time to Neutrophil and Platelet Engraftment

  151 days

Study Arms (1)

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. * Patients will remain on ruxolitinib for 1 year after transplant, at which point ruxolitinib will be tapered and discontinued. * Dose escalation will be permitted in patients with splenomegaly or myelofibrosis related symptoms.

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

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 criteria or secondary myelofibrosis as defined by the IWG-MRT criteria.
• Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria (Appendix G) OR
• Intermediate-1 risk disease with one of the following additional unfavorable features known to impact the survival adversely
• Red cell transfusion dependency
• Unfavorable Karyotype
• Platelet count ≤100 x 10\^9/L
• Presence of a high risk molecular marker associated with worsened overall survival (ASXL1, EZH2, IDH1/2, SRSF2, U2AF1, p53)
• 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 (HLA-A, B, DR) fully matched related donor
• /8 (HLA-A, B, DR, C) fully 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

You may not qualify if:

• Prior history of progressive multifocal leukoencephalopathy (PML)
• Concomitant receipt of St. John's Wort
• Hypersensitivity to any JAK inhibitor, including ruxolitinib, fedratinib, or any other 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)
• Patients with uncontrolled infection (patients with stable controlled infections such as hepatitis B or HIV patients with undetectable viral load on antiviral treatment would be eligible). Patients who are actively ill and require hospitalization to treat an infection will be excluded.
• History of another malignancy within 5-years of date of enrollment except those who have received definitive treatment. Definitive treatment will be defined as the use of surgery, chemotherapy or radiation for the treatment of a malignancy, which susbsquently has no evidence of disease after 2 years or \<10% probably of recurrence after 1 year. In addition, patients with history of the following are eligible:
• basal cell or squamous cell carcinoma of skin
• Polycythemia Vera or Essential Thrombocythemia
• ductal carcinoma in situ (DCIS)
• superficial bladder cancer
• prostatic intraepithelial neoplasia (PIN)
• 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

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

Limitations and Caveats

Single arm study

Results Point of Contact

• Title: Gabriela Hobbs
• Organization: MGH

ghobbs@mgb.org

617721124

Study Officials

• Gabriela Hobbs, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• 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 9, 2026
• Results First Posted: July 9, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)