A Phase I Study of LEE011 in Children and Adolescents with Malignant Rhabdoid Tumors and Neuroblastoma

Full Title
A Phase I, Multi-center, Open-label Study of LEE011 in Patients with Malignant Rhabdoid Tumors and Neuroblastoma (Novartis CLEE011X2102)
Disease Status

The primary purpose of this study is to find the highest dose of the investigational drug LEE011 that can be safely given in children and adolescents with malignant rhabdoid tumors and neuroblastoma that persist despite prior treatment. LEE011 has been evaluated in adults; this is the first study assessing its use in children. The drug is taken orally (by mouth).

LEE011 blocks the activity of two enzymes called cyclin D kinases 4 and 6 (CDK4/6). These enzymes are part of a pathway known to regulate cell growth. Blocking the activity of CDK 4/6 prevents some cancer cells from being able to make a new copy of their DNA. As a result, they cannot divide and may enter into a process called programmed cell death. Researchers hope LEE011 will slow the growth of cancer cells and lead to their death.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • This study is open to patients ages 12 months to 21 years (inclusive).
  • Patients must have a malignant rhabdoid tumor or neuroblastoma that has continued to grow despite standard therapy or for which no standard therapy exists.
  • Patients must have recovered from the serious side effects of prior therapies before entering the study.

For more information about this study and to inquire about eligibility, please contact Dr. Shakeel Modak at 212-639-7623.

Pediatric Brain Tumors: CNS Cancer