drugset
drugset / Trial / NCT01125800

A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB

NCT01125800 ↗

Phase 1/2 Completed 76 enrolled Novartis Pharmaceuticals
Non-randomizedSingle-groupOpen-labelTreatment

Summary

Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway. Phase II study is to assess preliminary efficacy in both adult and pediatric patients with recurrent or refractory MB.

Timeline

Start
2011-02
Primary completion
2014-10
Completion
2014-10

Drugs

EvaluationDrugModalityDoseRoute
Subject Sonidegib Small molecule 233 mg/m2 Oral
Subject Sonidegib Small molecule 372 mg/m2 Oral
Subject Sonidegib Small molecule 425 mg/m2 Oral
Subject Sonidegib Small molecule 680 mg/m2 Oral
Subject Sonidegib Small molecule 800 mg/m2 Oral

Indications

astrocytoma (excluding glioblastoma) hepatoblastoma medulloblastoma neuroblastoma rhabdomyosarcoma