NCT01747876

Study of Safety and Efficacy in Patients With Malignant Rhabdoid Tumors (MRT) and Neuroblastoma

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 1 Year – 21 Years
• Healthy volunteers: Not accepted

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 was 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 was also to have assessed the safety, tolerability, PK and preliminary evidence of antitumor activity of LEE011 in patients with MRT or neuroblastoma.

Detailed description

Due to lack of efficacy, enrollment in the study was stopped at the end of dose escalation (sites were notified of the early enrollment halt on 7-Aug-2014) and the dose-expansion part was not conducted. Due to halted enrollment and/or lack of complete responses (CR) and partial responses (PR), efficacy analysis was only performed in terms of TTP for the patients treated during the dose-escalation part at the maximum tolerated dose (MTD) and recommended dose expansion (RDE).

Conditions

• Malignant Rhabdoid Tumors (MRT)
• Neuroblastoma

Interventions

Type	Name	Description
DRUG	LEE011	LEE011 is a small molecule inhibitor of CDK4/6.

Timeline

• Start date: 2013-05-28
• Primary completion: 2017-06-29
• Completion: 2017-06-29
• First posted: 2012-12-12
• Last updated: 2019-11-22
• Results posted: 2019-02-28

Locations

13 sites across 5 countries: United States, Australia, France, Germany, United Kingdom

Regulatory

• FDA-regulated drug study