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Caspofungin Versus Fluconazole in Preventing Invasive Fungal Infections (IFI) in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia

C

Children's Oncology Group

Status and phase

Completed
Phase 3

Conditions

Untreated Childhood Myeloid Neoplasm
Childhood Acute Myeloid Leukemia With Maturation
Untreated Adult Acute Myeloid Leukemia
Neutropenia
Childhood Acute Myeloid Leukemia With Minimal Differentiation
Adult Acute Myelomonocytic Leukemia
Adult Acute Myeloid Leukemia in Remission
Adult Acute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A
Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11
Adult Acute Myeloid Leukemia With Minimal Differentiation
Adult Acute Myeloid Leukemia Without Maturation
Childhood Acute Myelomonocytic Leukemia
Childhood Acute Myeloid Leukemia Without Maturation
Adult Acute Monocytic Leukemia
Adult Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1
Recurrent Childhood Acute Myeloid Leukemia
Adult Acute Monoblastic Leukemia
Acute Myeloid Leukemia
Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11
Fungal Infection
Alkylating Agent-Related Acute Myeloid Leukemia
Childhood Acute Myeloid Leukemia in Remission
Childhood Acute Monocytic Leukemia
Childhood Acute Monoblastic Leukemia
Secondary Acute Myeloid Leukemia
Myeloid Neoplasm
Recurrent Adult Acute Myeloid Leukemia
Adult Acute Myeloid Leukemia With Maturation

Treatments

Other: Laboratory Biomarker Analysis
Drug: Caspofungin Acetate
Drug: Fluconazole

Study type

Interventional

Funder types

NETWORK
NIH

Identifiers

NCT01307579
S12-00316
CDR0000695748
UG1CA189955 (U.S. NIH Grant/Contract)
U10CA095861 (U.S. NIH Grant/Contract)
ACCL0933 (Other Identifier)
NCI-2011-02640 (Registry Identifier)
COG-ACCL0933

Details and patient eligibility

About

This randomized phase III trial compares the effectiveness of caspofungin to fluconazole in preventing invasive fungal infections in patients receiving chemotherapy for acute myeloid leukemia (AML). Antifungal prophylaxis is considered standard of care in children and adults with prolonged neutropenia after chemotherapy for AML however the ideal antifungal agent for prophylaxis in children is not known. Caspofungin has activity against yeast and some molds while fluconazole coverage is limited to just yeasts. Adult randomized trials suggest that agents with activity against yeasts and molds are more effective than those with just activity against yeasts. There are limited data to answer this comparative question in children. This study will establish much needed pediatric data to guide clinical decision making on optimal antifungal prophylaxis.

Full description

PRIMARY OBJECTIVES:

I. To determine if prophylaxis with caspofungin administered during periods of neutropenia following chemotherapy for acute myeloid leukemia (AML) is associated with a lower incidence of proven or probable invasive fungal infections (IFI) compared with fluconazole.

SECONDARY OBJECTIVES:

I. To determine if prophylaxis with caspofungin will result in a lower incidence of proven or probable cases of invasive aspergillosis (IA) compared with fluconazole. (Clinical) II. To determine if prophylaxis with caspofungin will result in improved survival compared to fluconazole. (Clinical) III. To determine if prophylaxis with caspofungin will result in less empiric antifungal therapy compared to fluconazole. (Clinical) IV. To determine the sensitivity, specificity, and positive and negative predictive value of biweekly galactomannan (GM) and beta-D glucan testing in diagnosing IFI. (Biological) V. To test the association between single nucleotide polymorphisms (SNPs) in genes involved in innate immunity and proven or probable IFI. (Biological) VI. To develop predictive models of IFI using SNP in genes involved in immunity and clinical covariates. (Biological)

OUTLINE: Patients are randomized to one of two treatment arms during their first chemotherapy course for AML.

ARM I: Patients receive caspofungin acetate intravenously (IV) over one hour once daily (QD) beginning within 24-72 hours following the last dose of chemotherapy for each course. and continuing until absolute neutrophil count (ANC) > 100-500/uL following the nadir or the next chemotherapy course begins.

ARM II: Patients receive fluconazole IV over 1-2 hours or orally (PO) QD beginning within 24-72 hours following the last dose of chemotherapy for each course.

Protocol prophylaxis was continued in both arms, until ANC increased to > 100-500/uL following the nadir or the next chemotherapy course began. Prophylaxis was given for all courses of planned AML chemotherapy or until the patient met one of the following off-protocol therapy criteria: development of proven or probable IFI according to institutional diagnosis, initiation of conditioning for hematopoietic cell transplantation, initiation of a new chemotherapy regimen for relapsed or refractory AML, refusal of further protocol therapy by patient, parent or guardian, or physician determines it is in the best interest of the patient.

Regardless of duration of prophylaxis, subjects in both arms are monitored for IFI until the earliest of the following criteria is met: two weeks after recovery of neutropenia following the last planned AML chemotherapy course, initiation of conditioning for hematopoietic cell transplantation, initiation of a new chemotherapy regimen for relapsed or refractory AML, withdrawal of consent for any further data submission, or death.

Patients were followed for overall survival up to two years from enrollment.

Enrollment

517 patients

Sex

All

Ages

3 months to 30 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Patients must have one of the following diagnoses and/or treatment plans:

    • Newly diagnosed de novo AML
    • First or subsequent relapse of AML
    • Secondary AML
    • Treatment with institutional standard AML therapy in those without AML (for example, myelodysplastic syndrome, bone marrow blasts > 5% or biphenotypia)
    • Note: Patients with a history of prolonged antifungal therapy (example, relapsed AML) are eligible
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender as follows:

    • =< 0.4 mg/dL (age 1 month to < 6 months)
    • =< 0.5 mg/dL (age 6 months to < 1 year)
    • =< 0.6 mg/dL (age 1 to < 2 years)
    • =< 0.8 mg/dL (age 2 to < 6 years)
    • =< 1 mg/dL (age 6 to < 10 years)
    • =< 1.2 mg/dL (age 10 to < 13 years)
    • =< 1.4 mg/dL (females age >= 13 years)
    • =< 1.5 mg/dL (males age 13 to < 16 years)
    • =< 1.7 mg/dL (males age >= 16 years)
  • Total bilirubin =< 1.5 x upper limit of normal (ULN) for age AND Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x ULN for age

  • All patients and/or their parents or legal guardians must sign a written informed consent

Exclusion criteria

  • Patients with the following diagnoses are not eligible:

    • Acute promyelocytic leukemia (APL)
    • Down syndrome
    • Juvenile myelomonocytic leukemia (JMML)
  • Patients with a documented history of invasive fungal infection (IFI) within the previous 30 days are not eligible

  • Patients with a history of echinocandin or fluconazole hypersensitivity are not eligible

  • Patients receiving treatment for an IFI are not eligible

  • Female patients of childbearing age must have a negative pregnancy test

  • Patients must agree to use an effective birth control method

  • Lactating patients must agree not to nurse a child while on this trial

Trial design

Primary purpose

Supportive Care

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

None (Open label)

517 participants in 2 patient groups

Arm I (caspofungin acetate)
Experimental group
Description:
Patients receive caspofungin acetate IV over one hour QD beginning within 24-72 hours following the last dose of chemotherapy for each course. and continuing until ANC \> 100-500/uL following the nadir or the next chemotherapy course begins.
Treatment:
Drug: Caspofungin Acetate
Other: Laboratory Biomarker Analysis
Arm II (fluconazole)
Active Comparator group
Description:
Patients receive fluconazole IV over 1-2 hours or PO QD beginning within 24-72 hours following the last dose of chemotherapy for each course.
Treatment:
Drug: Fluconazole
Other: Laboratory Biomarker Analysis

Trial documents
1

Trial contacts and locations

179

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Data sourced from clinicaltrials.gov

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