Combination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Promyelocytic Leukemia
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ClinicalTrials.gov Identifier: NCT00866918 |
Recruitment Status :
Active, not recruiting
First Posted : March 23, 2009
Results First Posted : April 4, 2017
Last Update Posted : March 25, 2022
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Condition or disease | Intervention/treatment | Phase |
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Childhood Acute Promyelocytic Leukemia (M3) Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies | Drug: arsenic trioxide Drug: mitoxantrone hydrochloride Other: diagnostic laboratory biomarker analysis Drug: idarubicin Drug: tretinoin Drug: cytarabine Drug: mercaptopurine tablet Drug: methotrexate | Phase 3 |

Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 108 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Risk Adapted Treatment of Newly Diagnosed Childhood Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide (Trisenox® IND# 103, 331) During Consolidation |
Actual Study Start Date : | March 9, 2009 |
Actual Primary Completion Date : | June 30, 2015 |

Arm | Intervention/treatment |
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Experimental: Standard Risk (WBC < 10000/uL)
See Detailed Description
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Drug: arsenic trioxide
Given IV
Other Names:
Drug: mitoxantrone hydrochloride Given IV
Other Names:
Other: diagnostic laboratory biomarker analysis Correlative studies Drug: idarubicin Given IV
Other Names:
Drug: tretinoin Given orally
Other Names:
Drug: cytarabine Given IT or IV
Other Names:
Drug: mercaptopurine tablet Given orally
Other Names:
Drug: methotrexate Given orally
Other Names:
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Active Comparator: High Risk (WBC > 10000/uL)
See Detailed Description
|
Drug: arsenic trioxide
Given IV
Other Names:
Drug: mitoxantrone hydrochloride Given IV
Other Names:
Other: diagnostic laboratory biomarker analysis Correlative studies Drug: idarubicin Given IV
Other Names:
Drug: tretinoin Given orally
Other Names:
Drug: cytarabine Given IT or IV
Other Names:
Drug: mercaptopurine tablet Given orally
Other Names:
Drug: methotrexate Given orally
Other Names:
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- Event-free Survival (EFS) [ Time Frame: At 3 years from study entry ]EFS - time from study entry until failure to achieve complete remission during consolidation, relapse, or death. For further clarification see definitions provided in the protocol.
- Hematologic Remission Rate [ Time Frame: End of consolidation, course 1: up to 5 months ]Proportion of patients in hematologic remission at end of consolidation, course 1 are reported.
- Hematologic, Molecular, and Cytogenetic Remission Rate [ Time Frame: End of consolidation, course 3; up to 7 months (for Standard Risk) or end of consolidation, course 4; up to 9 months (for High Risk) ]Proportion of patients in hematologic, molecular, and cytogenetic remission at end of consolidation, course 3 and 4 are reported. Patients were determined to be in remission by all three criteria.
- Overall Survival (OS) [ Time Frame: At 3 years from study entry ]OS - time from study entry to death.

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Ages Eligible for Study: | 2 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients must be newly diagnosed with a clinical diagnosis of acute promyelocytic leukemia initially by morphology (bone marrow or peripheral blood); bone marrow is highly preferred but in cases where marrow cannot be obtained at diagnosis, peripheral blood will be accepted; APL is considered a hematological emergency and treatment should be initiated as quickly as possible without waiting for molecular or cytogenetic/fluorescence in situ hybridization (FISH) confirmation; for patients who are unable to begin receiving ATRA in a timely manner following a presumed diagnosis of APL, consideration should be given to initiating ATRA and proceeding with treatment outside of the AAML0631 protocol; if the RQ-PCR results are known at the time of study enrollment, the patient must demonstrate PML-RARA and/or RARA-PML transcripts by RQ-PCR to be eligible; patients without evidence of APL by bone marrow or peripheral blood morphology but with isolated myeloid sarcoma (myeloblastoma; chloroma, including leukemia cutis) are eligible provided that the t(15;17) translocation is documented on either marrow or tumor tissue by cytogenetics, FISH, or PCR prior to study enrollment; in this situation, touch preps from the tumor site can be evaluated by FISH with PML-RARA probes; NOTE: A lumbar puncture is not required to be enrolled on study; if the diagnosis of APL is known or suspected, extreme caution must be exercised in performing a lumbar puncture during active coagulopathy; in addition a computed tomography (CT) or magnetic resonance imaging (MRI) should be considered to rule out the possibility of an associated chloroma if central nervous system (CNS) disease is suspected or proven; if CNS disease is documented, patients are still eligible
- No minimal performance status criteria
- The patient must not have received systemic definitive treatment for APL or other suspected leukemia, including cytotoxic chemotherapy, retinoids, or arsenic; prior therapy with corticosteroids, hydroxyurea, or leukopheresis will not exclude the patient; if a patient received intrathecal cytarabine prior to the diagnosis of APL being known, the patient will still be eligible as long as they meet all other eligibility requirements
Exclusion Criteria:
- Pregnant women or nursing mothers are excluded; treatment under this protocol would expose an unborn child to significant risks; patients should not be pregnant or plan to become pregnant while on treatment; women and men of reproductive potential should agree to use an effective means of birth control; there is an extremely high risk of fetal malformation if pregnancy occurs while on ATRA in any amount even for short periods
- Patients with a pre-existing prolonged QT Syndrome will not be eligible for this protocol due to the use of arsenic trioxide which can prolong the QT interval

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00866918

Principal Investigator: | John Gregory, MD | Children's Oncology Group |
Responsible Party: | Children's Oncology Group |
ClinicalTrials.gov Identifier: | NCT00866918 |
Other Study ID Numbers: |
AAML0631 NCI-2011-01904 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) ) CDR0000637184 ( Other Identifier: ClinicalTrials.gov ) AAML0631 ( Other Identifier: Children's Oncology Group ) AAML0631 ( Other Identifier: CTEP ) U10CA098543 ( U.S. NIH Grant/Contract ) |
First Posted: | March 23, 2009 Key Record Dates |
Results First Posted: | April 4, 2017 |
Last Update Posted: | March 25, 2022 |
Last Verified: | March 2022 |
Leukemia Leukemia, Promyelocytic, Acute Neoplasms by Histologic Type Neoplasms Leukemia, Myeloid, Acute Leukemia, Myeloid Cytarabine Methotrexate Mercaptopurine Mitoxantrone Idarubicin Arsenic Trioxide Tretinoin Abortifacient Agents, Nonsteroidal Abortifacient Agents |
Reproductive Control Agents Physiological Effects of Drugs Antimetabolites, Antineoplastic Antimetabolites Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Dermatologic Agents Enzyme Inhibitors Folic Acid Antagonists Immunosuppressive Agents Immunologic Factors Antirheumatic Agents Nucleic Acid Synthesis Inhibitors Antiviral Agents Anti-Infective Agents |