Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms

NCT ID: NCT02756572

Last Updated: 2021-11-08

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-09-22

Study Completion Date

2020-07-01

Brief Summary

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This clinical trial studies how well early stem cell transplantation works in treating patients with high-grade myeloid neoplasms that has come back after a period of improvement or does not respond to treatment. Drugs used in chemotherapy, such as filgrastim, cladribine, cytarabine and mitoxantrone hydrochloride, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor peripheral blood cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells. Early stem cell transplantation may result in more successful treatment for patients with high-grade myeloid neoplasms.

Detailed Description

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OUTLINE:

RE-INDUCTION CHEMOTHERAPY: Patients receive filgrastim subcutaneously (SC) on days 0-5, mitoxantrone hydrochloride intravenously (IV) over 60 minutes on days 1-3, cladribine IV over 2 hours on days 1-5, and cytarabine IV over 2 hours on days 1-5.

CONDITIONING REGIMEN: Beginning 14-60 days after re-induction chemotherapy, patients receive fludarabine phosphate IV over 30 minutes on days -6 to -2, melphalan IV on days -3 to -2, cyclosporine orally (PO) twice daily (BID) starting on day -3. Sirolimus PO BID starting on day -3 will be given to patients who have matched unrelated donors or mismatched unrelated donors. Patients \>55 years or with significant co-morbidities will only receive melphalan IV on day -2 and will also receive total body irradiation (TBI) on day -1 or day 0.

EARLY TRANSPLANT: Patients undergo allogeneic HCT after conditioning regimen on day 0.

GVHD PROPHYLAXIS: Patients with matched donors will receive mycophenolate mofetil PO three times daily (TID) on days 0-30, then twice a day (BID) until day 40; and cyclosporine PO BID on days -3 to 96, with a taper until day 150. Patients with matched unrelated donors also receive sirolimus PO BID on days -3 to 150, with a taper until day 180. Patients with mismatched unrelated donors will receive mycophenolate mofetil PO TID on days 0-30, then BID until day 100, with a taper until day 150; cyclosporine PO BID on days -3 to 150, then taper until day 180; and sirolimus BID PO days -3 to 180, then a taper until day 365.

After completion of study treatment, patients are followed up periodically.

Conditions

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Blasts 10 Percent or More of Bone Marrow Nucleated Cells Chronic Myelomonocytic Leukemia-2 High Grade Malignant Neoplasm Myelodysplastic Syndrome Myelodysplastic Syndrome With Excess Blasts-2 Myeloid Neoplasm Previously Treated Myelodysplastic Syndrome Recurrent Acute Myeloid Leukemia Refractory Acute Myeloid Leukemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment (chemotherapy, HCT)

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Group Type EXPERIMENTAL

Cladribine

Intervention Type DRUG

Given IV

Cyclosporine

Intervention Type DRUG

Given PO

Cytarabine

Intervention Type DRUG

Given IV

Filgrastim

Intervention Type BIOLOGICAL

Given SC

Fludarabine Phosphate

Intervention Type DRUG

Allogeneic Hematopoietic Stem Cell Transplantation

Intervention Type PROCEDURE

Undergo allogeneic hematopoietic stem cell transplantation

Laboratory Biomarker Analysis

Intervention Type OTHER

Correlative studies

Melphalan

Intervention Type DRUG

Given IV

Mitoxantrone Hydrochloride

Intervention Type DRUG

Given IV

Mycophenolate Mofetil

Intervention Type DRUG

Given PO

Questionnaire Administration

Intervention Type OTHER

Ancillary studies

Sirolimus

Intervention Type DRUG

Given PO

Total-Body Irradiation

Intervention Type RADIATION

Undergo TBI

Melphalan Hydrochloride

Intervention Type DRUG

Given IV

Interventions

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Cladribine

Given IV

Intervention Type DRUG

Cyclosporine

Given PO

Intervention Type DRUG

Cytarabine

Given IV

Intervention Type DRUG

Filgrastim

Given SC

Intervention Type BIOLOGICAL

Fludarabine Phosphate

Intervention Type DRUG

Allogeneic Hematopoietic Stem Cell Transplantation

Undergo allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

Laboratory Biomarker Analysis

Correlative studies

Intervention Type OTHER

Melphalan

Given IV

Intervention Type DRUG

Mitoxantrone Hydrochloride

Given IV

Intervention Type DRUG

Mycophenolate Mofetil

Given PO

Intervention Type DRUG

Questionnaire Administration

Ancillary studies

Intervention Type OTHER

Sirolimus

Given PO

Intervention Type DRUG

Total-Body Irradiation

Undergo TBI

Intervention Type RADIATION

Melphalan Hydrochloride

Given IV

Intervention Type DRUG

Other Intervention Names

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2-CdA 2CDA CdA Cladribina Leustat Leustatin Leustatine RWJ-26251 27-400 Ciclosporin CsA Cyclosporin Cyclosporin A Gengraf Neoral OL 27-400 Sandimmun Sandimmune SangCya .beta.-Cytosine arabinoside 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone 1-.beta.-D-Arabinofuranosylcytosine 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone 1-Beta-D-arabinofuranosylcytosine 1.beta.-D-Arabinofuranosylcytosine 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl- 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl- Alexan Ara-C ARA-cell Arabine Arabinofuranosylcytosine Arabinosylcytosine Aracytidine Aracytin Aracytine Beta-cytosine Arabinoside CHX-3311 Cytarabinum Cytarbel Cytosar Cytosine Arabinoside Cytosine-.beta.-arabinoside Cytosine-beta-arabinoside Erpalfa Starasid Tarabine PFS U 19920 U-19920 Udicil WR-28453 FILGRASTIM, LICENSE HOLDER UNSPECIFIED G-CSF Neupogen r-metHuG-CSF Recombinant Methionyl Human Granulocyte Colony Stimulating Factor rG-CSF Tevagrastim 2-F-ara-AMP 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)- Beneflur Fludara SH T 586 Allogeneic Hematopoietic Stem Cell HSCT Allogeneic Stem Cell Transplantation HSC Alanine Nitrogen Mustard CB-3025 L-PAM L-Phenylalanine Mustard L-sarcolysin L-Sarcolysin Phenylalanine mustard L-Sarcolysine Melphalanum Phenylalanine Mustard Phenylalanine nitrogen mustard Sarcoclorin Sarkolysin WR-19813 CL 232315 DHAD DHAQ Dihydroxyanthracenedione Dihydrochloride Mitoxantrone Dihydrochloride Mitoxantroni Hydrochloridum Mitozantrone Hydrochloride Mitroxone Neotalem Novantrone Onkotrone Pralifan Cellcept MMF AY 22989 RAPA Rapamune Rapamycin SILA 9268A WY-090217 Total Body Irradiation Whole-Body Irradiation 241286 3-(p-(bis(2-chloroethyl)amino)phenyl)-L-Alanine Hydrochloride Alkeran Alkerana Evomela

Eligibility Criteria

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Inclusion Criteria

* Relapsed or refractory high-grade myeloid neoplasms, defined as having a blast count of \>= 10% blasts at initial diagnosis; examples include excess blasts (EB)-2, with \>= 10% blasts at initial diagnosis, acute myeloid leukemia (AML) or chronic myelomonocytic leukemia (CMML-2); standard definitions of relapse will apply (i.e., characterized by \>= 5% abnormal blasts or blast equivalents as assessed by multiparameter flow cytometry or morphologic examination; peripheral blood blasts or blast equivalents; or extramedullary granulocytic sarcoma, per European LeukemiaNet \[ELN\] 2017 guidelines); bone marrow aspirate/biopsy will be accepted if performed outside University of Washington/Fred Hutchinson Cancer Research Center (UW/FHCRC); determination of disease status should occur within 30 days of signing informed consent

* R/R high-grade myeloid neoplasm following intensive induction chemotherapy; relapsed high-grade myeloid neoplasm: patients will be classified as relapsed if they have \>= 5% blasts after being in a complete remission (CR) following treatment for high-grade myeloid neoplasm; refractory high-grade myeloid neoplasm: patients may be classified as refractory if they have received at least one prior cycle of induction chemotherapy, whether with cladribine cytarabine mitoxantrone (GCLAM) or another regimen

\*\* Patients may have received up to two courses of intensive induction chemotherapy during initial treatment prior to enrollment on this protocol; for example, patients who have received two courses of granulocyte colony stimulating factor (G-CSF) GCLAM (or similar) chemotherapy, with most recent high-dose cytarabine-containing chemotherapy \> 6 months ago and CR lasting \> 6 months, will be eligible for this protocol; regimens "similar to GCLAM" would include cytarabine at doses of 1g/m\^2 for at least 5 doses; examples of regimens "similar to GCLAM" would be GCLA, fludarabine cytarabine granulocyte (FLAG), and FLAG-idarubicin (ida); however, patients who received more than two courses of GCLAM (or similar) chemotherapy, or patients who received two courses of GCLAM and had CR lasting \< 6 months, would not be eligible
* R/R high-grade myeloid neoplasm following less intensive induction chemotherapy. Patients who have received at least three cycles of treatment with a hypomethylating agent (HMA; such as azacitidine or decitabine) and still have \>= 10% blasts will be eligible for the study (they will be considered refractory); similarly, patients who have received three or more cycles of HMA therapy who have had a response (e.g., achieving CR with \< 5% blasts), but who then progress using standard definitions of relapse, will also be eligible (they will be considered relapsed)
* Potentially eligible for reduced intensity conditioning based on known organ function (formal organ function testing may occur after consent)
* Caregiver capable of providing post-HCT care
* Written informed consent


* Identified donor (see DONOR SELECTION below for further details)

* Matched related or unrelated (one allele mismatch in HLA-A, B, or C OK) donor according to institutional standards
* Unrelated volunteer donor who is mismatched with the recipient (i.e. 9/10 match)
* Caregiver capable of providing post-HCT care, who will be present once induction therapy with filgrastim, cladribine, cytarabine, mitoxantrone hydrochloride (GCLAM) begins
* Written informed consent for transplant
* Either bone marrow or peripheral blood is allowed

Exclusion Criteria

* Prior allogeneic HCT
* More than two prior courses of induction chemotherapy
* Relapse after minimal residual disease (MRD)-negative CR within 3 months of most recent GCLAM chemotherapy
* Low likelihood of being eligible for reduced intensity conditioning HCT based on known information

* Cardiac ejection fraction \< 40% or symptomatic coronary artery disease or uncontrolled arrhythmia, as assessed by multigated acquisition (MUGA) or transthoracic echocardiography (TTE) within previous 3 months and since the most recent anthracycline exposure
* Corrected diffusing capacity of the lungs for carbon monoxide (DLCOc) \< 40% or forced expiratory volume in 1 second (FEV1) \< 50%
* Estimated glomerular filtration rate (GFR) \< 40 ml/min
* Need for supplemental oxygen
* Direct bilirubin or alanine aminotransferase (ALT) \> 2 x upper limit of normal, unless these abnormalities are thought to be related to Gilbert's disease or leukemic infiltration of hepatic parenchyma
* Known human immunodeficiency virus (HIV) positivity
* Pregnant or nursing (to be confirmed with quantitative human chorionic gonadotropin \[HCG\] testing)
* Invasive solid tumor within 5 years; non-melanoma skin cancer or in situ malignancies are allowed
* Evidence of serious uncontrolled infection
* Eastern Cooperative Oncology Group (ECOG) of 3 or 4
* Donor specific antibodies against donor HLA-DQ or -DP
* Active bacterial, fungal or viral infections unresponsive to medical therapy
* Active leukemia in the central nervous system (CNS)
* HIV positive
* Cardiac ejection fraction \< 40% or symptomatic coronary artery disease or uncontrolled arrhythmia
* DLCOc \< 40% or FEV1 \< 50%
* Estimated GFR \< 40 ml/min
* Need for supplemental oxygen
* Direct bilirubin or ALT \> 2 x upper limit of normal, unless these abnormalities are thought to be related to Gilbert's disease or leukemic infiltration of hepatic parenchyma

DONOR SELECTION:

Identification of an appropriate donor will follow the general guidelines listed below.

* HLA-matched related or unrelated donor. Donors must be:

* Matched for HLA-A, B, C, DRB1 and DQB1 by high resolution typing
* Only a single allele disparity will be allowed for HLA-A, B, or C as defined by high resolution typing
* HLA-mismatched unrelated donor. Unrelated volunteer donors who are mismatched with the recipient within one of the following limitations will be permitted:

* Mismatch for one HLA class I antigen with or without an additional mismatch for one HLA-class I allele, but matched for HLA-DRB1 and HLA-DQ, OR
* Mismatched for two HLA class I alleles, but matched for HLA-DRB1 and HLA-DQ
* HLA class I HLA-A, -B, -C allele matched donors allowing for any one or two DRB1 and/or DQB1 antigen/allele mismatch

HLA-matching must be based on results of high resolution typing at HLA-A, -B, -C, -DRB1, and -DQ. If the patient is homozygous at the mismatch HLA class I locus or II locus, the donor must be heterozygous at that locus and one allele must match the patient (i.e., patient is homozygous A\*01:01 and donor is heterozygous A\*01:01, A\*02:01)
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

University of Washington

OTHER

Sponsor Role lead

Responsible Party

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Mary-Beth Percival

Assistant Professor, Division of Hematology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Mary-Elizabeth Percival

Role: PRINCIPAL_INVESTIGATOR

Fred Hutch/University of Washington Cancer Consortium

Locations

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Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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NCI-2016-00477

Identifier Type: REGISTRY

Identifier Source: secondary_id

9567

Identifier Type: OTHER

Identifier Source: secondary_id

P30CA015704

Identifier Type: NIH

Identifier Source: secondary_id

View Link

RG1016011

Identifier Type: OTHER

Identifier Source: secondary_id

9567

Identifier Type: -

Identifier Source: org_study_id