Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia

NCT ID: NCT00046930

Last Updated: 2023-07-05

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

449 participants

Study Classification

INTERVENTIONAL

Study Start Date

2002-09-17

Brief Summary

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RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Zosuquidar trihydrochloride, a modulator of multidrug resistance (MDR), may help daunorubicin and cytarabine kill more cancer cells by making cancer cells more sensitive to the drugs. It is not yet known whether daunorubicin and cytarabine are more effective with or without zosuquidar trihydrochloride in treating acute myeloid leukemia or anemia.

PURPOSE: This randomized phase III trial is studying how well giving zosuquidar trihydrochloride together with daunorubicin and cytarabine works compared to daunorubicin and cytarabine alone in treating older patients with newly diagnosed acute myeloid leukemia or anemia that has not responded to previous treatment.

Detailed Description

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OBJECTIVES:

* Compare the overall survival and progression-free survival of elderly patients with newly diagnosed acute myeloid leukemia, refractory anemia with excess blasts (RAEB) in transformation, or high-risk RAEB treated with daunorubicin and cytarabine with or without zosuquidar trihydrochloride.
* Compare the complete remission rate of patients treated with these regimens.
* Compare the toxicity of these regimens in these patients.
* Compare the systemic exposure of daunorubicin and cytarabine in patients treated with zosuquidar trihydrochloride vs placebo.

OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified according to age (60-69 years vs 70 years and over), disease (refractory anemia with excess blasts \[RAEB\] vs RAEB in transformation or acute myeloid leukemia \[AML\]), and disease type (de novo vs secondary). Patients are randomized to 1 of 2 treatment arms.

* Induction:

* Arm I: Patients receive daunorubicin via intravenous (IV) infusion over 10-15 minutes and zosuquidar trihydrochloride IV over 6 hours on days 1-3. Patients also receive cytarabine IV continuously on days 1-7.
* Arm II: Patients receive daunorubicin and cytarabine as in arm I. Patients also receive placebo IV over 6 hours on days 1-3.

Beginning on day 12, patients who achieve aplasia receive filgrastim (G-CSF) or sargramostim (GM-CSF) subcutaneously (SC) or IV daily until blood counts recover. Patients who have evidence of persistent AML are eligible to receive a second identical course of induction chemotherapy.

* Consolidation I (beginning within 8 weeks after documentation of complete remission \[CR\] or measurable remission \[MR\]): Patients who achieve a CR or MR receive cytarabine IV over 1 hour once or twice daily on days 1-6 and GM-CSF or G-CSF SC or IV beginning on day 7 and continuing until blood counts recover.
* Consolidation II: Patients who have maintained peripheral blood evidence of a remission receive daunorubicin, cytarabine, and zosuquidar trihydrochloride or placebo as in induction chemotherapy. Patients also receive GM-CSF or G-CSF SC or IV beginning on day 8 or after last cytarabine dose and continuing until blood counts recover.

Patients are followed monthly for 1 year, every 2 months for 1 year, every 3 months for 1 year, and then every 6 months for 2 years.

PROJECTED ACCRUAL: Approximately 450 patients (225 per treatment arm) accrued over 4.1 years.

Conditions

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Leukemia Myelodysplastic Syndromes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Zosuquidar

Induction treatment with daunorubicin, cytarabine and zosuquidar (details provided in Intervention section), followed by consolidation with Cytarabine (1500 mg/m2 every 12 hours for 6 days), then additional consolidation with the same regimen as received during induction.

Group Type EXPERIMENTAL

filgrastim

Intervention Type BIOLOGICAL

250 μg/m2/day by either intravenous or subcutaneous injection starting day 12, provided marrow aplasia is achieved, through recovery of absolute neutrophil count (ANC) to \> 500 cells/μl, sustained for 3 consecutive days. The dose may be rounded to the nearest vial size.

sargramostim

Intervention Type BIOLOGICAL

5 μg/kg/day by either intravenous or subcutaneous injection starting day 12, provided marrow aplasia is achieved, through recovery of absolute neutrophil count (ANC) to \> 500 cells/μl, sustained for 3 consecutive days. The dose may be rounded to the nearest vial size.

cytarabine

Intervention Type DRUG

100 mg/m²/day by continuous intravenous infusion for 7 days (days 1-7).

daunorubicin hydrochloride

Intervention Type DRUG

45 mg/m²/day by 10 - 15 minute intravenous infusion for 3 days (days 1, 2, and 3).

zosuquidar trihydrochloride

Intervention Type DRUG

Zosuquidar 550 mg/day by continuous intravenous infusion through a central venous catheter over approximately 6 hours on days 1, 2, and 3.

The infusion will begin approximately one hour prior to daunorubicin on days 1, 2 and 3.

Placebo

Induction treatment with daunorubicin, cytarabine and placebo (details provided in Intervention section), followed by consolidation with Cytarabine (1500 mg/m2 every 12 hours for 6 days), then additional consolidation with the same regimen as received during induction.

Group Type ACTIVE_COMPARATOR

filgrastim

Intervention Type BIOLOGICAL

250 μg/m2/day by either intravenous or subcutaneous injection starting day 12, provided marrow aplasia is achieved, through recovery of absolute neutrophil count (ANC) to \> 500 cells/μl, sustained for 3 consecutive days. The dose may be rounded to the nearest vial size.

sargramostim

Intervention Type BIOLOGICAL

5 μg/kg/day by either intravenous or subcutaneous injection starting day 12, provided marrow aplasia is achieved, through recovery of absolute neutrophil count (ANC) to \> 500 cells/μl, sustained for 3 consecutive days. The dose may be rounded to the nearest vial size.

cytarabine

Intervention Type DRUG

100 mg/m²/day by continuous intravenous infusion for 7 days (days 1-7).

daunorubicin hydrochloride

Intervention Type DRUG

45 mg/m²/day by 10 - 15 minute intravenous infusion for 3 days (days 1, 2, and 3).

Placebo

Intervention Type DRUG

Placebo 550 mg/day by continuous intravenous infusion through a central venous catheter over approximately 6 hours on days 1, 2, and 3.

The infusion will begin approximately one hour prior to daunorubicin on days 1, 2 and 3. Placebo consisted of a 1:1000 dilution of Infuvite, appropriately colored.

Interventions

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filgrastim

250 μg/m2/day by either intravenous or subcutaneous injection starting day 12, provided marrow aplasia is achieved, through recovery of absolute neutrophil count (ANC) to \> 500 cells/μl, sustained for 3 consecutive days. The dose may be rounded to the nearest vial size.

Intervention Type BIOLOGICAL

sargramostim

5 μg/kg/day by either intravenous or subcutaneous injection starting day 12, provided marrow aplasia is achieved, through recovery of absolute neutrophil count (ANC) to \> 500 cells/μl, sustained for 3 consecutive days. The dose may be rounded to the nearest vial size.

Intervention Type BIOLOGICAL

cytarabine

100 mg/m²/day by continuous intravenous infusion for 7 days (days 1-7).

Intervention Type DRUG

daunorubicin hydrochloride

45 mg/m²/day by 10 - 15 minute intravenous infusion for 3 days (days 1, 2, and 3).

Intervention Type DRUG

zosuquidar trihydrochloride

Zosuquidar 550 mg/day by continuous intravenous infusion through a central venous catheter over approximately 6 hours on days 1, 2, and 3.

The infusion will begin approximately one hour prior to daunorubicin on days 1, 2 and 3.

Intervention Type DRUG

Placebo

Placebo 550 mg/day by continuous intravenous infusion through a central venous catheter over approximately 6 hours on days 1, 2, and 3.

The infusion will begin approximately one hour prior to daunorubicin on days 1, 2 and 3. Placebo consisted of a 1:1000 dilution of Infuvite, appropriately colored.

Intervention Type DRUG

Other Intervention Names

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Neupogen, recombinant-methionyl human granulocyte-colony stimulating factor, granulocyte colony-stimulating factor, r-metHuG-CSF Granulocyte-macrophage colony stimulating factor, rHu GM-CSF, Leukine, NSC # 617589 Cytosar-U, Ara-C, Arabinosyl, cytosine arabinoside. Daunomycin, Rubidomycin, Cerubidine. Multi drug resistance modulator, MDR, LY335979 Baxter Infuvite Adult

Eligibility Criteria

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Inclusion Criteria

One of the following disorders:

* Acute myeloid leukemia (AML), defined as \>30% myeloblasts on the marrow aspirate or peripheral blood differential and any French-American-British (FAB) subtype except M3 (i.e., acute promyelocytic leukemia)
* Refractory anemia with excess blasts (RAEB), defined as 11-20% myeloblasts on bone marrow aspirate or peripheral blood differential, provided there are other criteria for high-risk disease
* Refractory anemia with excess blasts in transformation (RAEB-T), defined as 21-30% myeloblasts on bone marrow aspirate or peripheral blood differential
* Participants may have secondary AML
* Age greater than 60 years
* ECOG performance status of 0 to 3
* Total serum bilirubin \< 3 mg/dL
* Serum creatinine \< 2 mg/dL
* Cardiac ejection fraction of \> 45%

Exclusion Criteria

* Blastic transformation of chronic myelogenous leukemia
* CNS leukemia
* Prior chemotherapy for AML, with the exception of hydroxyurea
* For women: pregnant or breast feeding
* Other malignancy for which participant is currently receiving treatment
* Concurrent treatment with other colony-stimulating factors
Minimum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Eli Lilly and Company

INDUSTRY

Sponsor Role collaborator

Kanisa Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

Eastern Cooperative Oncology Group

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Larry D. Cripe, MD

Role: STUDY_CHAIR

Indiana University Melvin and Bren Simon Cancer Center

Locations

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CCOP - Mayo Clinic Scottsdale Oncology Program

Scottsdale, Arizona, United States

Site Status

Aurora Presbyterian Hospital

Aurora, Colorado, United States

Site Status

Boulder Community Hospital

Boulder, Colorado, United States

Site Status

Penrose Cancer Center at Penrose Hospital

Colorado Springs, Colorado, United States

Site Status

Porter Adventist Hospital

Denver, Colorado, United States

Site Status

Presbyterian - St. Luke's Medical Center

Denver, Colorado, United States

Site Status

St. Joseph Hospital

Denver, Colorado, United States

Site Status

Rose Medical Center

Denver, Colorado, United States

Site Status

CCOP - Colorado Cancer Research Program, Incorporated

Denver, Colorado, United States

Site Status

Swedish Medical Center

Englewood, Colorado, United States

Site Status

Sky Ridge Medical Center

Lone Tree, Colorado, United States

Site Status

Hope Cancer Care Center at Longmont United Hospital

Longmont, Colorado, United States

Site Status

St. Mary-Corwin Regional Medical Center

Pueblo, Colorado, United States

Site Status

North Suburban Medical Center

Thornton, Colorado, United States

Site Status

University of Florida Shands Cancer Center

Gainesville, Florida, United States

Site Status

Baptist Cancer Institute - Jacksonville

Jacksonville, Florida, United States

Site Status

Mayo Clinic - Jacksonville

Jacksonville, Florida, United States

Site Status

Watson Clinic, LLC

Lakeland, Florida, United States

Site Status

H. Lee Moffitt Cancer Center and Research Institute at University of South Florida

Tampa, Florida, United States

Site Status

MBCCOP-Medical College of Georgia Cancer Center

Augusta, Georgia, United States

Site Status

Hematology and Oncology Associates

Chicago, Illinois, United States

Site Status

Robert H. Lurie Comprehensive Cancer Center at Northwestern University

Chicago, Illinois, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Decatur Memorial Hospital Cancer Care Institute

Decatur, Illinois, United States

Site Status

Evanston Northwestern Health Care - Evanston Hospital

Evanston, Illinois, United States

Site Status

Regional Cancer Center at Memorial Medical Center

Springfield, Illinois, United States

Site Status

Carle Cancer Center at Carle Foundation Hospital

Urbana, Illinois, United States

Site Status

Indiana University Cancer Center

Indianapolis, Indiana, United States

Site Status

Methodist Cancer Center at Methodist Hospital

Indianapolis, Indiana, United States

Site Status

McFarland Clinic, P.C.

Ames, Iowa, United States

Site Status

Siouxland Hematology-Oncology Associates

Sioux City, Iowa, United States

Site Status

St. Luke's Regional Medical Center

Sioux City, Iowa, United States

Site Status

Cancer Center of Kansas - Chanute

Chanute, Kansas, United States

Site Status

Cancer Center of Kansas - Dodge City

Dodge City, Kansas, United States

Site Status

Cancer Center of Kansas - Kingman

Kingman, Kansas, United States

Site Status

Southwest Medical Center

Liberal, Kansas, United States

Site Status

Cancer Center of Kansas - Newton

Newton, Kansas, United States

Site Status

Pratt Cancer Center of Kansas

Pratt, Kansas, United States

Site Status

Cancer Center of Kansas - Salina

Salina, Kansas, United States

Site Status

Cancer Center of Kansas - Wellington

Wellington, Kansas, United States

Site Status

Associates in Womens Health

Wichita, Kansas, United States

Site Status

Cancer Center of Kansas, P.A.

Wichita, Kansas, United States

Site Status

Cancer Center of Kansas, P.A. - Wichita

Wichita, Kansas, United States

Site Status

CCOP - Wichita

Wichita, Kansas, United States

Site Status

Via Christi Cancer Center at Via Christi Regional Medical Center

Wichita, Kansas, United States

Site Status

Wesley Medical Center

Wichita, Kansas, United States

Site Status

Cancer Center of Kansas - Winfield

Winfield, Kansas, United States

Site Status

Tufts - New England Medical Center

Boston, Massachusetts, United States

Site Status

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status

Baystate Regional Cancer Program at D'Amour Center for Cancer Care

Springfield, Massachusetts, United States

Site Status

CCOP - Michigan Cancer Research Consortium

Ann Arbor, Michigan, United States

Site Status

St. Joseph Mercy Cancer Center at St. Joseph Mercy Hospital

Ann Arbor, Michigan, United States

Site Status

Borgess Medical Center

Kalamazoo, Michigan, United States

Site Status

Bronson Methodist Hospital

Kalamazoo, Michigan, United States

Site Status

West Michigan Cancer Center

Kalamazoo, Michigan, United States

Site Status

Fairview Ridges Hospital

Burnsville, Minnesota, United States

Site Status

Mercy and Unity Cancer Center at Mercy Hospital

Coon Rapids, Minnesota, United States

Site Status

Fairview Southdale Hospital

Edina, Minnesota, United States

Site Status

Mercy and Unity Cancer Center at Unity Hospital

Fridley, Minnesota, United States

Site Status

Virginia Piper Cancer Institute at Abbott-Northwestern Hospital

Minneapolis, Minnesota, United States

Site Status

Hubert H. Humphrey Cancer Center at North Memorial Medical Center

Robbinsdale, Minnesota, United States

Site Status

Mayo Clinic Cancer Center

Rochester, Minnesota, United States

Site Status

CCOP - Metro-Minnesota

Saint Louis Park, Minnesota, United States

Site Status

Park Nicollet Clinic

Saint Louis Park, Minnesota, United States

Site Status

United Hospital

Saint Paul, Minnesota, United States

Site Status

Ridgeview Medical Center

Waconia, Minnesota, United States

Site Status

University Medical Center of Southern Nevada

Las Vegas, Nevada, United States

Site Status

CCOP - Southern Nevada Cancer Research Foundation

Las Vegas, Nevada, United States

Site Status

CCOP - Northern New Jersey

Hackensack, New Jersey, United States

Site Status

Booker Cancer Center at Riverview Medical Center

Red Bank, New Jersey, United States

Site Status

NYU Cancer Institute at New York University Medical Center

New York, New York, United States

Site Status

Albert Einstein Cancer Center at Albert Einstein College of Medicine

The Bronx, New York, United States

Site Status

Leo W. Jenkins Cancer Center at Pitt County Memorial Hospital

Greenville, North Carolina, United States

Site Status

Aultman Hospital Cancer Center at Aultman Health Foundation

Canton, Ohio, United States

Site Status

Ireland Cancer Center at University Hospitals of Cleveland and Case Western Reserve University

Cleveland, Ohio, United States

Site Status

MetroHealth's Cancer Care Center at MetroHealth Medical Center

Cleveland, Ohio, United States

Site Status

St. Luke's Hospital Cancer Center

Bethlehem, Pennsylvania, United States

Site Status

Geisinger Medical Center

Danville, Pennsylvania, United States

Site Status

Penn State Cancer Institute at Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Abramson Cancer Center of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Hillman Cancer Center at University of Pittsburgh Cancer Institute

Pittsburgh, Pennsylvania, United States

Site Status

Guthrie Medical Center - Sayre

Sayre, Pennsylvania, United States

Site Status

Hematology and Oncology Associates

Scranton, Pennsylvania, United States

Site Status

Geisinger Medical Group

State College, Pennsylvania, United States

Site Status

Geisinger Wyoming Valley Medical Center

Wilkes-Barre, Pennsylvania, United States

Site Status

Sioux Valley Hospital and University of South Dakota Medical Center

Sioux Falls, South Dakota, United States

Site Status

University of Virginia Cancer Center

Charlottesville, Virginia, United States

Site Status

Mary Babb Randolph Cancer Center at West Virginia University Hospitals

Morgantown, West Virginia, United States

Site Status

Gundersen Lutheran Cancer Center at Gundersen Lutheran Medical Center

La Crosse, Wisconsin, United States

Site Status

University of Wisconsin Comprehensive Cancer Center

Madison, Wisconsin, United States

Site Status

Marshfield Clinic - Marshfield Center

Marshfield, Wisconsin, United States

Site Status

Rambam Medical Center

Haifa, , Israel

Site Status

Countries

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United States Israel

References

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Cripe LD, Li X, Litzow M, et al.: A randomized, placebo-controlled, double blind trial of the MDR modulator, zosuquidar, during conventional induction and post-remission therapy for Pts > 60 years of age with newly diagnosed acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (HR-MDS): ECOG 3999. [Abstract] Blood 108 (11): A-423, 2006.

Reference Type RESULT

Foran JM, Sun Z, Lai C, Fernandez HF, Cripe LD, Ketterling RP, Racevskis J, Luger SM, Paietta E, Lazarus HM, Zhang Y, Bennett JM, Levine RL, Rowe JM, Litzow MR, Tallman MS. Obesity in adult acute myeloid leukemia is not associated with inferior response or survival even when dose capping anthracyclines: An ECOG-ACRIN analysis. Cancer. 2023 Aug 15;129(16):2479-2490. doi: 10.1002/cncr.34807. Epub 2023 Apr 25.

Reference Type DERIVED
PMID: 37185873 (View on PubMed)

Cripe LD, Uno H, Paietta EM, Litzow MR, Ketterling RP, Bennett JM, Rowe JM, Lazarus HM, Luger S, Tallman MS. Zosuquidar, a novel modulator of P-glycoprotein, does not improve the outcome of older patients with newly diagnosed acute myeloid leukemia: a randomized, placebo-controlled trial of the Eastern Cooperative Oncology Group 3999. Blood. 2010 Nov 18;116(20):4077-85. doi: 10.1182/blood-2010-04-277269. Epub 2010 Aug 17.

Reference Type DERIVED
PMID: 20716770 (View on PubMed)

Other Identifiers

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E3999

Identifier Type: OTHER

Identifier Source: secondary_id

U10CA021115

Identifier Type: NIH

Identifier Source: secondary_id

View Link

CDR0000257122

Identifier Type: -

Identifier Source: org_study_id

NCT00046046

Identifier Type: -

Identifier Source: nct_alias

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