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Reduced Intensity Conditioning

Stem Cell Transplant for Myelofibrosis

Phase 2

Recruiting

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

Meeting criteria for intermediate-1, intermediate-2, or high-risk disease by DIPSS-plus scoring system

Patient must be a potential hematopoietic stem cell transplant candidate

Must not have

Availability of HLA-identical or 1-allele-mismatched related donor or HLA 10 of 10 matched unrelated donor

Significant renal or hepatic impairment including cirrhosis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying the outcomes of using a JAK inhibitor prior to reduced intensity haploidentical transplantation for the treatment of primary or secondary myelofibrosis.

Who is the study for?

This trial is for adults over 18 with primary or secondary myelofibrosis who may benefit from a stem cell transplant. They should have been on a JAK inhibitor for at least 8 weeks, have good organ function, and not be pregnant or HIV positive. Ideal candidates don't have severe liver disease, uncontrolled infections, or previous allogeneic transplants.

What is being tested?

The study tests if taking a JAK inhibitor before undergoing reduced intensity haploidentical transplantation can lower graft failure in myelofibrosis patients. It includes pre-treatment with drugs like Cyclophosphamide and Fludarabine followed by stem cell transplantation.

What are the potential side effects?

Potential side effects include reactions to medications such as Cyclophosphamide (nausea, hair loss), Tacrolimus (kidney problems, high blood pressure), and risks associated with stem cell transplantation like infection risk increase and graft-versus-host disease.

Eligibility Criteria

Inclusion Criteria

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

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

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I am considered a candidate for a stem cell transplant.

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I have been diagnosed with primary or secondary myelofibrosis.

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I am older than 18 years.

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

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My kidneys are functioning well.

Exclusion Criteria

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

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I have a donor who is a perfect or near-perfect match for me.

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I have severe kidney or liver problems.

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I have an infection that is not under control.

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My blood cancer has progressed significantly.

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I do not have any uncontrolled infections.

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

Probability of primary and secondary graft failure

Side effects data

From 2014 Phase 3 trial • 87 Patients • NCT00075478

23%

Blood/Bone marrow

11%

Cardiovascular

Pulmonary

Gastrointestinal

Hepatic

Graft versus host disease with infection and organ failure

Dermatology/Skin

respiratory failure

Hemorrhage

subdural hematoma

thrombosis

Renal/Genitourinary

Metabolic/Laboratory

100%

80%

60%

40%

20%

Study treatment Arm

Arm II (TBI, Transplant, GVHD Prophylaxis)

Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort II (JAK inhibitor, conditioning, GVHD prophylaxis)Experimental Treatment16 Interventions

JAK INHIBITOR THERAPY: Patients receive a JAK inhibitor at least 8 weeks prior to the start of HCT conditioning through day -4 before transplantation. Additionally, patients receive a JAK inhibitor following transplantation beginning day 5 through 9-12 months after transplant. CONDITIONING: Patients receive melphalan IV over 1 hour on day -5, fludarabine IV over 30-60 minutes on days -5 to -2, and undergo TBI on day -1 or day -1 and day 0. TRANSPLANT: Patients receive peripheral blood stem cell infusion on day 0. GVHD PROPHYLAXIS: Patients then receive cyclophosphamide IV over 3 hours on days 3-4, tacrolimus IV beginning day 5 then PO for about 6 months, mycophenolate mofetil PO BID or TID beginning day 5 for 6 weeks, and G-CSF SC beginning day 7 until neutrophil recovery is \> 1,500/mm\^3. All patients undergo MRI, CT, bone marrow biopsy and aspiration and blood sample collection throughout the trial. Patients also undergo ECHO or MUGA on the trial.

Group II: Cohort I (JAK inhibitor, conditioning, GVHD prophylaxis)Experimental Treatment16 Interventions

JAK INHIBITOR THERAPY: Patients receive a JAK inhibitor at least 8 weeks prior to the start of HCT conditioning through day -4 before transplantation. CONDITIONING: Patients receive melphalan IV over 1 hour on day -5, fludarabine IV over 30-60 minutes on days -5 to -2, and undergo TBI on day -1 or day -1 and day 0. TRANSPLANT: Patients receive peripheral blood stem cell infusion on day 0. GVHD PROPHYLAXIS: Patients then receive cyclophosphamide IV over 3 hours on days 3-4, tacrolimus IV beginning day 5 then PO for about 6 months, mycophenolate mofetil PO BID or TID beginning day 5 for 6 weeks, and G-CSF SC beginning day 7 until neutrophil recovery is \> 1,500/mm\^3. All patients undergo MRI, CT, bone marrow biopsy and aspiration and blood sample collection throughout the trial. Patients also undergo ECHO or MUGA on the trial.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Computed Tomography

2017

Completed Phase 2

~2790

Magnetic Resonance Imaging

2017

Completed Phase 3

~1180