Safety Study of CPX-351 in Children With Relapsed Leukemia or Lymphoma
Condition: Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Burkitt Lymphoma; Diffuse Large Cell Lymphoma; Gray Zone Lymphoma; Lymphoblastic Lymphoma; Anaplastic Large Cell Lymphoma; Hodgkin Lymphoma
MeSH terms: Burkitt Lymphoma; Lymphoma; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Lymphoma, Non-Hodgkin; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Large-Cell, Anaplastic
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to test the safety of a study drug called CPX-351. This drug has
      been tested in adults but not yet in children and adolescents. This study tests different
      doses of the drug to see which dose is safer in children and adolescents.

      Patients who have blood cancer are being asked to take part in this study . Blood cancers may
      include leukemia and lymphoma. Patients able to be in this study have already been treated
      with standard chemotherapy for their disease and the disease is still growing or has come
      back.

      CPX-351 is a drug that is not yet approved by the United States Food and Drug Administration
      (FDA) and is only used in research studies like this one. CPX-351 is made up of two
      chemotherapy drugs that patients may have already received called cytarabine and daunorubicin
      that are now packaged together.

      Another purpose of this study is to collect blood samples for special research studies.
      Researchers want to study how much of the CPX-351 is in the body over time. These studies are
      call pharmacokinetic studies or PK studies for short. PK studies require the collection of
      several blood samples before and after participants are given the study drug.
    

Detailed description: 
      Cytarabine in combination with an anthracycline is a frequently used chemotherapy platform
      for both newly diagnosed and relapsed/refractory acute myeloid leukemia (AML) and other
      hematologic malignancies. Synergistic antitumor activity has been demonstrated between
      cytarabine and daunorubicin that is dependent upon the ratio of the drugs with the best
      therapeutic effect observed with a cytarabine to daunorubicin ratio of 5:1 in in vitro and in
      vivo models. CPX-351 is a liposomal preparation of cytarabine and daunorubicin that maintains
      this therapeutic drug ratio 24 hours post infusion. The altered biodistribution from
      encapsulation may result in a greater therapeutic effect in patients with relapsed
      hematologic malignancies and demonstrate greater tolerability than non-liposomal cytarabine
      and daunorubicin.

      This is a single institution phase-I pilot study that aims to assess the pharmacokinetics,
      toxicity and tolerability of CPX-351 in pediatric and young adults with relapsed/refractory
      hematologic malignancies. Subjects will receive a single course of CPX-351 administered on
      Days 1, 3, and 5. The study will first open to children in a dose exploration phase, and then
      be available to an expanded cohort, which will be open to children and young adults once a
      tolerable dose has been determined.
    

Intervention name(s): CPX-351
Intervention type(s): Drug
Intervention description(s): Comparison of Different doses of drug
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Relapsed acute myeloid leukemia; non-therapy related acute myeloid leukemia; Refractory acute myeloid leukemia; Untreated acute myeloid leukemia; Lymphoma; Leukemia