ADVL0921: A Phase II Study of MLN8237 (IND# 102984), a Selective Aurora A Kinase Inhibitor in Children With Recurrent / Refractory Solid Tumors and Leukemia

Why are we doing this research?

This phase II trial is studying the side effects of and how well Alisertib works in treating young patients with relapsed or refractory solid tumors or leukemia.

Who can participate?

Diagnosis of 1 of the following:

  • Histologically confirmed malignant solid tumor at original diagnosis or relapse
  • Neuroblastoma
  • Rhabdomyosarcoma (RMS)
  • Osteosarcoma
  • Ewing sarcoma/peripheral primitive neuroectodermal tumor
  • Non-RMS soft tissue sarcoma
  • Hepatoblastoma
  • Malignant germ cell tumor
  • Wilms tumor
  • Histologically confirmed leukemia recurrent or refractory to ≥2 prior induction or treatment regimens
  • Acute lymphoblastic leukemia 
  •  > 25% blasts in the bone marrow (M3 bone marrow), excluding known CNS disease
  • Acute myeloid leukemia
  • ≥5 % blasts in the bone marrow (M2/M3 bone marrow), excluding known CNS disease
  • Radiographically measurable disease for solid tumors
  • Patients with neuroblastoma who do not have measurable disease but have iodine-123 metaiodobenzylguanidine (¹²³ I-MIBG)-positive lesions allowed

None of the following qualify as measurable disease:

  • Malignant fluid collections (e.g., ascites, pleural effusions)
  • Bone marrow infiltration
  • Lesions detected by nuclear medicine studies (e.g., bone, gallium, or PET scans)
  • Elevated tumor markers in plasma or cerebrospinal fluid
  • Previously irradiated lesions that have not demonstrated clear progression after radiotherapy
  • No CNS disease (leukemia patients)


  • Cancer - Kidney Tumors
  • Cancer - Leukemia and Lymphoma
  • Cancer - Liver Tumors


  • Female
  • Male

What will happen in the study?

This is a multicenter study. Patients are stratified according to type of tumor (solid tumor and measurable disease vs neuroblastoma with MIBG-positive lesions vs neuroblastoma with bone marrow involvement vs AML vs ALL). Patients receive oral aurora A kinase inhibitor Alisertib once daily on days 1-7. Treatment repeats every 21 days for up to 35 courses in the absence of disease progression or unacceptable toxicity. Plasma samples are collected from all patients at baseline and periodically during course 1 for pharmacokinetic and other studies. After completion of study therapy, patients are followed up for 5 years.


Cincinnati Children’s Hospital Medical Center
Division of Hematology/Oncology
3333 Burnet Ave., Cincinnati, OH 45229-3039
Phone: 513-636-2799