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Protocol No. AAML1331

Principal Investigator DeSantes, Kenneth

Phase III

Age Group Both

Scope National

Sponsor Type Institutional; National

Title A Phase III Study for Patients with Newly Diagnosed Acute Promyelocytic Leukemia (APL) using Arsenic Trioxide and All-Trans Retinoic Acid

Objective Standard treatment for acute promyelocytic leukemia (APL) involves high doses of a common class of chemotherapy drugs called anthracyclines. High doses of anthracyclines are known to cause long-term side effects, especially to the heart. Study doctors are interested in finding an effective treatment for APL that reduces the potential for long-term side effects. A clinical study has shown that a drug called arsenic trioxide, given with other standard chemotherapy drugs, works well in the treatment for APL in adults. Arsenic trioxide has also been evaluated in small groups of children with APL. These studies have determined a dose of arsenic trioxide that is well tolerated with other chemotherapy drugs. The main goal of this study is to find out if completely removing or reducing the amount of anthracycline chemotherapy used in standard APL treatment, and adding arsenic trioxide, will reduce some of the long-term side effects while maintaining a good cure rate.

Treatment All subjects will be assigned to a risk group to determine their treatment. The term 'risk' refers to the chance of the cancer coming back after treatment. This would happen even if you do not take part in a research study. A blood test will show if you have high or standard risk APL. Treatment is divided into the following stages: Induction, Consolidation Cycle 1, Consolidation Cycle 2, MRD Positive Consolidation (not for all subjects), Consolidation Cycle 3 and Consolidation Cycle 4. The term 'MRD' means minimal residual disease and it refers to a special test that is done to detect small amounts of disease in the bone marrow. There is a difference in treatment between the subjects with high risk and standard risk APL on this study. Subjects with high risk APL will also receive the drugs idarubicin and dexamethasone during Induction. Subjects with standard risk APL will not receive these drugs.

Description Drugs that are part of the treatment regimen in this protocol are given in a variety of ways. Tretinoin is given by mouth, arsenic trioxide, cytarabine,and mitoxantrone are given intravenously.

Key Eligibility
  • Patient must be ≥ 12 months and < 22 years of age at first diagnosis of APL
  • Patients must be newly diagnosed with a clinical diagnosis of APL
  • Patients may receive up to a maximum of 5 days of pre-treatment with ATRA prior to administration of protocol therapy
  • Treatment with hydroxyurea, corticosteroids (any route) and intrathecal cytarabine prior to beginning protocol directed therapy is allowed
  • Patients with secondary APL are excluded
  • Patients with isolated myeloid sarcoma (myeloblastoma, chloroma, including leukemia cutis) but without evidence of APL by bone marrow or peripheral blood morphology are excluded
  • Patients who have received treatment with any other cytotoxic chemotherapy prior to beginning protocol therapy are excluded
  • Female patients who are pregnant are excluded
  • Lactating females who plan to breastfeed their infants are excluded

Applicable Disease Sites Leukemia

Therapies Involved Cytotoxic Chemotherapy

Drugs Involved Trisenox (arsenic trioxide); ara-c (cytarabine); arsenic trioxide; atra (all-trans-retinoic acid); cytarabine; cytosar (cytarabine); cytosine arabinoside (cytarabine); decadron (dexamethasone); dexamethasone; droxia (hydroxyurea); hdmtx (methotrexate); hu (hydroxyurea); hydrea (hydroxyurea); hydrocortisone; hydroxyurea; id (idarubicin); ida (idarubicin); idamycin (idarubicin); idarubicin; it-mtx (methotrexate); leucovorin; leukovorin (leucovorin); lv (leucovorin); methotrexate; mitoxantrone; mtx (methotrexate); novantrone (mitoxantrone); oh-urea (hydroxyurea); solu-cortef (hydrocortisone); wellcovorin (leucovorin)

Status Open

Participating Institutions Gundersen Health System; UW Hospital and Clinics