1002 / 1266
Randomized Phase II C2201 Study Design
Hh-pathway activated relapsed MB in children and adults
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)
Hh+ rMB:
Children and adults
who have relapsed
after RT; and
children who are not
candidates for RT
Randomize
2:1
Arm 1: (n=46)
LDE225
@RP2D
Arm 2: (n=23)
TMZ
QD x5 every
28 days
N=69
RT = radiotherapy
PD
D/C due to
AE or PD
or other
reasons or
death
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