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Hematopoietic Stem Cell Transplantation

Chemo/Radiation Therapy + Stem Cell Transplant for Myelofibrosis

Phase 1

Recruiting

Led By Monzr M Al Malki

Research Sponsored by City of Hope Medical Center

Eligibility Criteria Checklist

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

Must have

DONOR: Infectious disease screening performed within 30 days prior to stem cell mobilization per federal guidelines and is: Seronegative for HIV 1+2 antibody (Ab) and/or HIV polymerase chain reaction (PCR), human T-cell leukemia virus (HTLV) I/II Ab, hepatitis B virus surface antigen (HBsAg), hepatitis B virus surface antibody (HBcAb), hepatitis C virus (HCV) Ab, Negative rapid plasma reagin (RPR) for syphilis

No active infections

Must not have

Evidence of severe portal hypertension with evidence of decompensation either with bleeding varices, large volume ascites, or hepatic encephalopathy

DONOR: Has undergone solid organ, stem cell, bone marrow or blood transplantation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

All Individual Drugs Already Approved

Approved for 20 Other Conditions

No Placebo-Only Group

Summary

This trial is testing a chemo/radiation therapy followed by a stem cell transplant to treat secondary myelofibrosis.

Who is the study for?

This trial is for patients with secondary myelofibrosis who are fit (Karnofsky performance >=70%), have adequate organ function, and no severe pulmonary or liver conditions. Donors must be a genetic half-match without certain infections or health risks, and not pregnant if female of childbearing potential.

What is being tested?

The trial tests a treatment combining chemotherapy drugs (Melphalan, Mycophenolate Mofetil, Tacrolimus, Cyclophosphamide, Fludarabine), total body irradiation, and donor blood stem cell transplant to see how well it treats secondary myelofibrosis.

What are the potential side effects?

Potential side effects include damage to organs from radiation and chemotherapy such as nausea, fatigue, infection risk increase due to immune suppression; complications from the stem cell transplant like graft failure; and reactions related to the infusion.

Eligibility Criteria

Inclusion Criteria

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

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I am a donor with recent tests showing no HIV, HTLV, hepatitis B or C, and syphilis.

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I do not have any ongoing infections.

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My kidneys are functioning well.

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My myelofibrosis is severe enough to need a transplant, according to DIPSS-plus.

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My donor is a half-match for me based on genetic testing.

Exclusion Criteria

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

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I have severe liver issues with symptoms like bleeding, fluid buildup, or confusion.

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I have had a transplant involving solid organ, stem cell, bone marrow, or blood.

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I am not currently on any experimental drugs or undergoing chemotherapy, immunosuppression, or radiation.

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I, as a donor, have health issues that prevent me from undergoing growth factor therapy and leukapheresis safely.

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I have liver cirrhosis.

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My cancer has spread to my brain or spinal cord.

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I am a donor without risk factors for complications from donation procedures.

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I am not pregnant or have not been breastfeeding for the last 6 months.

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My bone marrow test shows more than 15% blasts, or more than 5% if I previously had AML.

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I am HIV positive or have active hepatitis B or C.

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I have severe pulmonary hypertension confirmed by heart tests.

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I currently have an active infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 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

Incidence of adverse events

Incidence of unacceptable toxicity

Secondary study objectives

Cumulative incidence of acute graft versus host disease (GvHD)

Cumulative incidence of chronic graft versus host disease GvHD

Cumulative incidence of relapse/progression

+7 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 20 Other Conditions

This treatment demonstrated efficacy for 20 other conditions.

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 (combination chemotherapy, TBI, HCT)Experimental Treatment9 Interventions

Patients receive melphalan IV over 30 minutes on day -5, fludarabine IV over 30-60 minutes on days -5 to -2. Patients undergo TBI on day -1 and HCT on day 0. Patients receive cyclophosphamide IV over 1-2 hours on days 3 and 4. Starting on day 5, patients receive tacrolimus IV then PO for 6 months followed by a taper, mycophenolate mofetil PO TID until day 35, and G-CSF IV daily until absolute neutrophil count \> 1,500/mm\^3 for 3 consecutive days. Treatment continues in the absence of disease progression or unexpected toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

FDA approved

Tacrolimus

FDA approved

Allogeneic Hematopoietic Stem Cell Transplantation

2012

Completed Phase 2

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