Conditions: Medulloblastoma Rhabdomyosarcoma Neuroblastoma Hepatoblastoma Glioma Astrocytoma
Keywords: Recurrent, refractory, medulloblastoma, rhabdomyosarcoma, neuroblastoma, hepatoblastoma, astrocytoma, children, pediatric, hedgehog pathway inhibitor, adult
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 76
Healthy volunteers: no

Brief 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.

Detailed description:

Arms:
arm 1: Pediatric dose. arm 2: Pediatric dose. arm 3: Pediatric dose. arm 4: Pediatric dose. arm 5: Adult dose

Interventions: intervention 1: LDE225
Intervention descriptions: intervention 1: LDE225/sonidegib capsules were supplied to the Investigators at dose strengths of 50 mg, 100 mg, 200 mg, and 250 mg.

Protocol document: