Brain Tumours

Randomized Phase II C2201 Study Design Hh-pathway activated relapsed MB in children and adults

N=69

D/C due to AE or PD or other reasons or death

Arm 1: (n=46) LDE225 @RP2D Arm 2: (n=23) TMZ QD x5 every 28 days

Hh+ rMB: Children and adults who have relapsed after RT; and children who are not candidates for RT

Randomize 2:1

PD

RT = radiotherapy

Primary endpoints: ORR Secondary endpoints: DoR, PFS, safety, OS, QoL

Randomization stratified by age (adults/children) Patients on TMZ may cross-over to LDE225 at progression. Patients not previously treated with RT will be analyzed as a separate subgroup. Safety data will be analyzed by age (children/adults)

LDE225C2201 Pivotal Medulloblastoma | Confidential | Business Use Only