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Eligibility
for people ages 1 year to 21 years
Location
at San Francisco, California and other locations
Dates
study started
estimated completion:
Principal Investigator

Description

Summary

LEE011 is a small molecule inhibitor of CDK4/6. LEE011 has demonstrated in vitro and in vivo activity in both tumor models. The primary purpose of this study is to determine the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) in pediatric patients and to delineate a clinical dose to be used in future studies. This study will also assess the safety, tolerability, PK and preliminary evidence of antitumor activity of LEE011 in patients with MRT or neuroblastoma.

Official Title

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

Keywords

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

Eligibility

You can join if…

Open to people ages 1 year to 21 years

  • Confirmed diagnosis of MRT or, neuroblastoma or in dose escalation part, other tumors with documented evidence of D-cyclin-CDK4/6-INK4a-Rb pathway abnormalities (dose escalation part only),
  • 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 50.

You CAN'T join if...

  • 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.

Locations

  • Children's Healthcare of Atlanta Dept of Oncology
    Atlanta, Georgia, 30342, USA
  • Dana Farber Cancer Institute SC-7
    Boston, Massachusetts, 02215, USA
  • Memorial Sloan Kettering Cancer Center Dept of Onc
    NY, New York, 10065, USA
  • Cincinnati Children's Hospital Medical Center Dept of Oncology
    Cincinnati, Ohio, 45229, USA
  • St. Jude's Children's Research Hospital Dept of Oncology
    Memphis, Tennessee, 38105, USA
  • Novartis Investigative Site
    Perth, Western Australia, Australia
  • Novartis Investigative Site
    Lyon Cedex, 69373, France
  • Novartis Investigative Site
    Paris, 75231, France
  • Novartis Investigative Site
    Villejuif Cedex, 94805, France
  • Novartis Investigative Site
    Koeln, Nordrhein-Westfalen, 50937, Germany
  • Novartis Investigative Site
    Augsburg, 86156, Germany
  • Novartis Investigative Site
    Sutton, Surrey, SM2 5PT, United Kingdom

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Novartis Pharmaceuticals
ID
NCT01747876
Phase
Phase 1
Lead Scientist
Katherine Matthay
Study Type
Interventional
Last Updated
February 2017