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Reduced Intensity Haploidentical Transplantation for the Treatment of Primary or Secondary Myelofibrosis

ConditionsPrimary MyelofibrosisSecondary Myelofibrosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-70
SponsorFred Hutchinson Cancer Center

About this trial

This initial cohort of this phase II trial studied the outcomes of using a JAK inhibitor prior to reduced intensity haploidentical (Haplo) transplantation for the treatment of primary or secondary myelofibrosis (MF). The primary risk of using Haplo HCT in patients with MF is graft failure. In the first cohort, all patients engrafted. There were no instances of graft failure. However, a large number of patients did have graft versus host disease as a complication of their transplant. JAK inhibitors have since been approved for the indication of graft versus host disease treatment. And we are also using them for graft versus host disease prevention in a study of MF patients with sibling and unrelated donors. Therefore, we are opening a new cohort of the current study using the JAK inhibitor prior to, during and after Haplo transplant. Our goal is to decrease graft versus host disease in patients receiving a Haplo MF transplant without increasing the risk of graft failure.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

PART 1: JAK INHIBITOR ADMINISTRATION INCLUSION CRITERIA

Age between 18 and 70 years

Diagnosis of primary myelofibrosis (PMF) as defined by the 2016 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

Patients meeting the criteria for intermediate-1, intermediate-2 or high-risk disease by the Dynamic International Prognostic Scoring System (DIPSS)-plus scoring system (DIPSS may be used if all data from DIPSS are not available)

Disqualifiers

PART 1: JAK INHIBITOR ADMINISTRATION EXCLUSION CRITERIA

Patients who have known hypersensitivity to JAK inhibitors

Clinical or laboratory evidence of significant renal or hepatic impairment including cirrhosis

Active uncontrolled infection

Trial design

Design model

Single group

Treatments tested in this trial

  • Cyclophosphamide

    Drug

    Given IV

  • JAK Inhibitor

    Drug

    Given PO

  • Fludarabine

    Drug

    Given IV

  • Recombinant Granulocyte Colony-Stimulating Factor

    Biological/Vaccine

    Given SC

  • Melphalan

    Drug

    Given IV

  • Mycophenolate Mofetil

    Drug

    Given PO

  • Peripheral Blood Stem Cell Transplantation

    Procedure/Surgery

    Given IV

  • Tacrolimus

    Drug

    Given IV and PO

  • Total-Body Irradiation

    Radiation

    Undergo TBI

  • Computed Tomography

    Procedure/Surgery

    Undergo CT

  • Magnetic Resonance Imaging

    Procedure/Surgery

    Undergo MRI

  • Bone Marrow Biopsy

    Procedure/Surgery

    Undergo bone marrow biopsy and aspiration

  • Bone Marrow Aspiration

    Procedure/Surgery

    Undergo bone marrow biopsy and aspiration

  • Biospecimen Collection

    Procedure/Surgery

    Undergo blood sample collection

  • Echocardiography Test

    Procedure/Surgery

    Undergo ECHO

  • Multigated Acquisition Scan

    Procedure/Surgery

    Undergo MUGA

Treatment groups

20 Participants
are divided into 2 treatment groups
Group A: Cohort I (JAK inhibitor, conditioning, GVHD prophylaxis)Experimental treatment 16 interventions
Group B: Cohort II (JAK inhibitor, conditioning, GVHD prophylaxis)Experimental treatment 16 interventions

Trial outcomes

Primary outcomes

1

Probability of primary and secondary graft failure

Primary graft failure is defined as failure to achieve an absolute neutrophil count of \> 500/ul by 42 days after bone marrow or peripheral blood stem cell transplantation. Secondary graft failure is defined as cytopenias after initial engraftment, with (a) donor chimerism of \< 5% or (b) falling donor chimerism with intervention such as second transplant or donor lymphocyte infusion or (c) patient death due to cytopenias, and fall in donor chimerism, even if chimerism is \> 5%. Exclusion criteria for diagnosis of graft failure are (a) disease relapse, (b) graft-versus-host disease, and (c) other causes of cytopenias such as, viral infection and drug toxicity.

Time frame
Up to 5 years

Secondary outcomes

1

Incidence of severe (grade 3 or 4) cytokine release syndrome

Time frame
Up to 5 years
2

Non-relapse mortality (NRM)

Time frame
Day 100
3

NRM

Time frame
1 year
4

Overall survival

Time frame
1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Fred Hutchinson Cancer Center

Lead sponsor

Source ClinicalTrials.gov
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