← Back to Search

Stem Cell Transplantation

Pre-Transplant JAK Inhibitor for Myelofibrosis

Phase 2

Waitlist Available

Led By Rachel B. Salit

Research Sponsored by Fred Hutchinson Cancer Research Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients meeting the criteria for intermediate-1, intermediate-2 or high-risk disease by the Dynamic International Prognostic Scoring System (DIPSS) or DIPSS-plus scoring system

Diagnosis of primary MF (PMF) as defined by the 2008 World Health Organization classification system or diagnosis of secondary MF as defined by the International Working Group (IWG) for Myeloproliferative Neoplasms Research and Treatment criteria

Must not have

Patients without an HLA-identical or 1-allele-mismatched related donor or unrelated donor or umbilical cord blood units that meet transplant criteria

Evidence of human immunodeficiency virus (HIV) infection or known HIV positive serology

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether a JAK inhibitor can improve transplant outcomes for patients with myelofibrosis by being given before the transplant.

Who is the study for?

This trial is for patients with primary or secondary myelofibrosis, a type of bone marrow cancer. Participants should have certain risk levels by DIPSS scores, good physical function (Karnofsky >= 70), adequate kidney and liver function, no severe lung issues, and not be on supplemental oxygen. They must understand the study and consent to participate. Pregnant or breastfeeding individuals, those with HIV or uncontrolled infections, prior transplants are excluded.

What is being tested?

The trial tests if taking a JAK inhibitor drug called ruxolitinib before receiving stem cells from a donor can improve outcomes for myelofibrosis patients. The treatment aims to reduce symptoms like spleen enlargement and help the new stem cells work better in producing healthy blood cells after transplant.

What are the potential side effects?

Potential side effects include risks associated with ruxolitinib such as infection risk increase due to immune system suppression, liver problems, anemia (low red blood cell count), bruising or bleeding easily due to low platelets. Transplant-related side effects may include graft-versus-host disease where donor cells attack patient's body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My condition is considered intermediate-1, intermediate-2, or high-risk.

Select...

I have been diagnosed with primary or secondary myelofibrosis according to specific medical guidelines.

Select...

My kidney function, measured by creatinine clearance, is good.

Select...

My donor is a near-perfect match for my transplant.

Select...

I am able to care for myself but may not be able to do active work.

Select...

I do not have severe liver problems or diseases that affect my liver function.

Select...

I may be eligible for a stem cell transplant.

Select...

I have been on ruxolitinib for at least 8 weeks and can continue it until 4 days before my transplant.

Select...

My bone marrow donor is a sibling who is a complete or near-complete match.

Select...

I do not use supplemental oxygen.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I don't have a closely matched donor for a transplant.

Select...

I am HIV positive.

Select...

I do not have any ongoing serious infections.

Select...

I have had a transplant from another person.

Select...

I do not have any uncontrolled infections.

Select...

I have a history of HIV infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

2-year Overall Survival (OS) in Patients With Myelofibrosis (MF) Who Receive Treatment With a JAK Inhibitor Followed by an Allogeneic Transplant

Secondary study objectives

Non-relapse Mortality (NRM)

Number of Patients Who Experienced Primary Graft Failure/Rejection

Number of Patients Who Experienced Secondary Graft Failure/Poor Graft Function

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (ruxolitinib, transplant)Experimental Treatment12 Interventions

Patients receive a ruxolitinib and undergo myeloablative or reduced-intensity conditioning followed by transplant and GVHD prophylaxis; see detailed description.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Allogeneic Hematopoietic Stem Cell Transplantation

2012

Completed Phase 2

~1240

Busulfan

2008

Completed Phase 4

~1710