NCT00609739 · REGISTRY CONDITION SEARCH

Cytosine Arabinoside and Mitoxantrone for Patients With Juvenile Myelomonocytic Leukemia Receiving Repeat Stem Cell Transplantation

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE1, PHASE2
Status at capture: TERMINATED
Registry last update: 2017-12-28

RATIONALE: Giving chemotherapy drugs, such as cytarabine and mitoxantrone, before a donor stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine, methotrexate, and methylprednisolone before or after transplant may stop this from happening. PURPOSE: This phase I/II trial is studying the side effects and best way to give high-dose cytarabine together with mitoxantrone in treating patients with juvenile myelomonocytic leukemia undergoing a second donor stem cell transplant.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Disease-free Survival

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outcome · POSTED

Patients With Regimen-Related Toxicity

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outcome · POSTED

Patients With Graft-Versus-Host-Disease

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outcome · POSTED

Patients Who Relapsed

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

eligibility Criteria: Inclusion Criteria: * Patients age 0-18 with juvenile myelomonocytic leukemia (JMML) who have relapsed or have residual disease after allogeneic HCT. Residual disease is defined as failure to eradicate original disease without prior documentation of remission. Relapse is defined as reappearance of i) leukocytosis with absolute monocytosis \>1 x 10\^8/L, ii) presence of immature myeloid cells in the peripheral circulation in two consecutive bone marrow specimens taken at least one month apart, or iii) presence of clonal cytogenetic abnormality. The diagnosis of relapse will be supported by the return of an abnormal cytogenetic marker (if present at diagnosis) or the presence of host cells by RFLP or other method. * Patients should be at least 6 months from first hematopoietic cell transplant (HCT) if clinically stable. (If JMML is rapidly progressive, second HCT may be performed earlier). * Adequate major organ function including: * Cardiac: ejection fraction ≥45% * Pulmonary: FEV \>50%, DLCO \>50% * Renal: creatinine clearance ≥40 mL/min * Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites) * Karnofsky performance status ≥70% or Lansky score ≥50% * Written informed consent. Exclusion Criteria: * Active uncontrolled infection within one week of HCT.
healthy Volunteers: false
maximum Age: 18 Years
sex: ALL
std Ages: CHILD
ADULT

Treatment arms and interventions

arm Groups

description
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
intervention Names
Drug: cyclosporine
Drug: cytarabine
Drug: filgrastim
Drug: methotrexate
Drug: methylprednisolone
Drug: mitoxantrone hydrochloride
Procedure: allogeneic bone marrow transplantation
Procedure: umbilical cord blood transplantation
Drug: Cis-Retinoic acid
label
Cytarabine + Mitoxantrone
type
EXPERIMENTAL

interventions

arm Group Labels
Cytarabine + Mitoxantrone
description
Patients will receive CSA therapy beginning on day -3, with a taper commencing on day +60 (unless GVHD) and ending on day +90. For patients \>40 kg with normal renal function (creatinine \<1.3 mg/dL), the initial dose will be 2.5 mg/kg intravenously (IV) over 2 hours every 12 hours. For children \<40 kg, the initial dose will be 2.5 mg/kg IV over 2 hours every 8 hours.
name
cyclosporine
other Names
CSA
type
DRUG
arm Group Labels
Cytarabine + Mitoxantrone
description
3000 mg/m\^2 intravenously (IV) over 2 hours x 2 (i.e. total 6000 mg/m\^2/day) on days -9 through -4.
name
cytarabine
other Names
Ara-C
type
DRUG
arm Group Labels
Cytarabine + Mitoxantrone
description
Patients with absolute neutrophil count (ANC) \<0.2 x 10\^8/L on day 21 may receive G-CSF at 5 mcg/kg/day. G-CSF will be continued until ANC ≥2.5 x 10\^8/L for two consecutive days. As the malignant cell population of JMML is known to be hypersensitive to GM-CSF, this cytokine will not be given to these patients.
name
filgrastim
other Names
G-CSF
type
DRUG
arm Group Labels
Cytarabine + Mitoxantrone
description
MTX will be administered to recipients of non-genotypically identical BMT. MTX will be administered at a dose of 15 mg/m\^2 (based on adjusted ideal body weight) intravenously (IV) on day +1 and at a dose of 10 mg/m\^2 IV on days +3, +6, and +11.
name
methotrexate
other Names
MTX
type
DRUG
arm Group Labels
Cytarabine + Mitoxantrone
description
Recipients of UCB will receive methylprednisolone 2 mg/kg/day from day +5 to +19 at a dose of 1 mg/kg twice a day (bid) with a 10% taper every week thereafter.
name
methylprednisolone
other Names
Medrol
type
DRUG
arm Group Labels
Cytarabine + Mitoxantrone
description
10 mg/m\^2 over 30 minutes intravenously (IV) on days -9 through -7.
name
mitoxantrone hydrochloride
other Names
Mitoxantrone
type
DRUG
arm Group Labels
Cytarabine + Mitoxantrone
description
Donor marrow will be collected in the usual sterile manner with a collection goal of 2.0 \>10\^8/kg recipient weight. Infused on Day 0.
name
allogeneic bone marrow transplantation
type
PROCEDURE
arm Group Labels
Cytarabine + Mitoxantrone
description
Umbilical cord blood (UCB) will be cryopreserved prior to transplantation. Cord blood units will be selected for transplantation according to current University of Minnesota Department of Blood and Marrow Transplantation Guidelines.
name
umbilical cord blood transplantation
type
PROCEDURE
arm Group Labels
Cytarabine + Mitoxantrone
description
Post-Transplant Cis-Retinoic Acid (CRA) Therapy - CRA will be given at a dosage of 100 mg/m\^2/day by mouth in a single daily dose starting on day +60 and continuing until 1 year after transplant.
name
Cis-Retinoic acid
other Names
isotretinoin
type
DRUG

Study design

allocation

NON_RANDOMIZED

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 1
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Number of patients who were free of disease and alive at 1 year.
measure
Disease-free Survival
time Frame
1 year

secondary Outcomes

description
Number of patients with adverse events related to treatment.
measure
Patients With Regimen-Related Toxicity
time Frame
Up to 30 Days Post Study Treatment
description
Number of patients who exhibited acute and/or chronic graft-versus-host disease.
measure
Patients With Graft-Versus-Host-Disease
time Frame
Up to 30 Days Post Study Treatment
description
Number of patients whose disease relapsed.
measure
Patients Who Relapsed
time Frame
1 Year

Full study description

brief Summary: RATIONALE: Giving chemotherapy drugs, such as cytarabine and mitoxantrone, before a donor stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine, methotrexate, and methylprednisolone before or after transplant may stop this from happening. PURPOSE: This phase I/II trial is studying the side effects and best way to give high-dose cytarabine together with mitoxantrone in treating patients with juvenile myelomonocytic leukemia undergoing a second donor stem cell transplant.
detailed Description: OBJECTIVES: Primary * To determine the incidence of 1-year disease-free survival in patients with juvenile myelomonocytic leukemia and who is undergoing a repeat stem cell transplantation. Secondary * To evaluate the incidence of regimen-related toxicity. * To evaluate the incidence of acute and chronic graft-versus-host-disease. * To evaluate the incidence of relapse. OUTLINE: * Preparative cytoreductive therapy: Patients receive high-dose cytarabine IV over 2 hours on days -9 to -4 and mitoxantrone hydrochloride IV over 30 minutes on days -9 to -7. * Allogeneic hematopoietic stem cell transplantation (HSCT): Patients undergo HSCT on day 0. Patients undergoing umbilical cord blood transplantation receive methylprednisolone (as graft failure prophylaxis) IV twice daily on days 5 to 19 followed by a taper every other day thereafter until day 25. * Graft-versus-host-disease (GVHD) prophylaxis: Patients receive cyclosporine IV over 2 hours every 8-12 hours or orally twice daily beginning on day -3 and continuing until day 50, followed by a taper to day 90, in the absence of GVHD. Patients undergoing nongenotypically identical bone marrow transplantation also receive methotrexate IV on day 1 beginning 24 hours after completion of stem cell infusion and on days 3, 6, and 11. * Post-transplantation isotretinoin therapy: Patients receive oral isotretinoin once daily beginning on day 60 and continuing until 1 year after HSCT. Patients undergo bone marrow sample collection on day 21, day 60, day 100, at 6 months, and at 1 year for chimerism studies. Patients also undergo blood sample collection periodically to monitor peripheral blood counts for immune reconstitution. After completion of study treatment, patients are followed on day 21, day 100, at 6 months, and at 1 year.

Source references

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice