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A Phase I, Multi-center, Open-label Study of LEE011 in Patients With Malignant Rhabdoid Tumors and Neuroblastoma

Phase 1
1 Year
21 Years
Open (Enrolling)
Malignant Rhabdoid Tumors (MRT), Neuroblastoma

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Trial Information

A Phase I, Multi-center, Open-label Study of LEE011 in Patients With Malignant Rhabdoid Tumors and Neuroblastoma

Inclusion Criteria:

- Confirmed diagnosis of MRT or neuroblastoma, that has progressed despite standard
therapy or for which no effective standard therapy exists.

- Patients with CNS disease should be on stable doses of steroids for at least 7 days
prior to first dose of LEE011 with no plans for escalation.

- In expansion part, patients must have at least one measurable disease as defined by
RECIST v1.1.

- Patients must have a Lansky (≤ 16 years) or Karnofsky (> 16 years) score of at least

Exclusion Criteria:

- Prior history of QTc prolongation or QTcF > 450 ms on screening ECG.

- Patients with the following laboratory values during screening:

- Serum creatinine > 1.5 x upper limit of normal (ULN) for age

- Total bilirubin >1.5 x ULN for age

- Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) > 3 x
ULN for age; aspartate aminotransferase (AST)/serum glutamic oxaloacetic
transaminase(SGOT) > 3 x ULN for age except in patients with tumor involvement
of the liver who must have AST/SGOT and ALT/SGPT ≤ 5 x ULN for age. For the
purpose of this study, the ULN for SGPT/ALT is 45 U/L.

- Patients who are currently receiving treatment with agents that are metabolized
predominantly through CYP3A4/5 and have a narrow therapeutic window and/or agents
that are known strong inducers or inhibitors CYP3A4/5 are prohibited. In particular,
enzyme-inducing antiepileptic drugs (EIAEDs).

- Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality that may interfere with the interpretation of study results, and in the
judgment of the investigator would make the patient inappropriate for the study.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Incidence rate of dose limiting toxicities (DLTs)

Outcome Description:

Estimate the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of LEE011 as a single agent when administered orally to patients with malignant rhabdoid tumors (MRTs) or neuroblastoma

Outcome Time Frame:

cycle 1 = 28 days (from the time of first dose)

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

May 2013

Completion Date:

November 2014

Related Keywords:

  • Malignant Rhabdoid Tumors (MRT), Neuroblastoma
  • Phase 1
  • pediatric
  • CDK4/6 inhibitor
  • dose escalation
  • malignant rhabdoid tumors
  • MRT
  • neuroblastoma
  • Neuroblastoma
  • Rhabdoid Tumor



University of California San Francisco Dept of Pediatic Oncology San Francisco, California  94101
Children's Healthcare of Atlanta Dept of Oncology Atlanta, Georgia  30342
Dana Farber Cancer Institute SC-7 Boston, Massachusetts  02115
Seattle Cancer Care Alliance/Fred Hutchinson Cancer Research Dept Oncology Seattle, Washington  98109-1023
Cincinnati Children's Hospital Medical Center Dept of Oncology Cincinnati, Ohio  45229-3039
St. Jude's Children's Research Hospital Dept of Oncology Memphis, Tennessee  38105-2794
Memorial Sloan Kettering Cancer Center Dept of Onc New York, New York  10021
Children's Hospital of Philadelphia Dept of Oncology Philadelphia, Pennsylvania  19104-4399