A dose finding and safety study of oral LDE225 in paediatric patients.
Research type
Research Study
Full title
A Phase I Study of LDE225 in Paediatric Patients with Recurrent or Refractory Medulloblastoma, or Other Tumours Potentially Dependent on Hedgehog Signalling Pathway.
IRAS ID
57282
Contact name
Darren Hargrave
Sponsor organisation
Novartis Pharma Services AG
Eudract number
2010-019348-37
ISRCTN Number
Not Known
Research summary
The purpose of this phase 1 study is to assess the safety, tolerability, Pharmacokinetic and preliminary evidence of antitumour activity of LDE225 in children with medulloblastoma, rhabdomyosarcoma, neuroblastoma, hepatoblastoma, high-grade glioma, or osteosarcoma, in order to determine the maximum tolerated dose (MTD) in children, and to delineate a clinical dose to be used in future studies. Activation of the hedgehog signalling pathway has been linked to several paediatric tumours; rhabdomyosacroma, neuroblastoma, hepatoblastoma or astrocytoma.LDE225 is a new investigational drug that reduces the effectiveness of the hedgehog pathway by binding to the smoothened (Smo) regulator and therefore may inhibit tumour growth.LDE225 has been studied extensively in the lab and is currently being evaluated in 44 patients in a first time in human phase I study where a favourable tolerability profile and anti-tumour activity has so far been observed along with a pharmacologically active dose range for adults. This is an open label trial in children aged greater than or equal to 12 months and <18 years old and will take place in 2 parts, a dose escalation phase where the recommended and or maximum tolerated dose of LDE225 will be established and a safety expansion phase where patients will receive the recommended or maximum tolerated dose to further gain confidence in the safety and tolerability of this dose.
REC name
London - Riverside Research Ethics Committee
REC reference
10/H0706/90
Date of REC Opinion
16 Mar 2011
REC opinion
Further Information Favourable Opinion