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

A Phase I/II Study of LDE225 in Pediatric Patients With Recurrent or Refractory Medulloblastoma or Other Tumors Potentially Dependent on the Hedgehog-signaling Pathway and Adult Patients With Recurrent or Refractory Medulloblastoma

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 76 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 12 Months – 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Medulloblastoma
• Rhabdomyosarcoma
• Neuroblastoma
• Hepatoblastoma
• Glioma
• Astrocytoma

Interventions

Type	Name	Description
DRUG	LDE225	LDE225/sonidegib capsules were supplied to the Investigators at dose strengths of 50 mg, 100 mg, 200 mg, and 250 mg.

Timeline

• Start date: 2011-02-01
• Primary completion: 2014-10-01
• Completion: 2014-10-01
• First posted: 2010-05-18
• Last updated: 2017-03-20
• Results posted: 2016-03-02

Locations

14 sites across 6 countries: United States, Australia, Canada, France, Italy, United Kingdom