Activation of the Sonic Hedgehog (SHH) Pathway Clinical Trial
Official title:
An International, Randomized, Open-label Phase I/II Study of Vismodegib in Combination With Temozolomide Versus Temozolomide Alone in Adult Patients With Recurrent or Refractory Medulloblastomas Presenting an Activation of the Sonic Hedgehog (SHH) Pathway
The purpose of this study is to evaluate the safety of vismodegib in combination with
temozolomide (primary objective - phase I) and to estimate the efficacy of vismodegib in
combination with temozolomide in adult patients with recurrent, progressive, or refractory
medulloblastomas to standard therapy measured by the 6-month progression-free rate (phase
II).
This study is an open-label Phase I/II, international, randomized.
38 patients will be included in the study.
Secondary objectives are :
phase I : to collect preliminary results on the 6-month progression-free rate of the
combination vismodegib + temozolomide
PHASE II
To estimate in the two study arms:
- the objective response rate (Complete response + Partial Response according to WHO
criteria) after 6 months of treatment
- the duration of treatment response
- the best overall response obtained during the study
- the progression-free survival (PFS)
- the time to progression (TTP)
- the time to treatment failure (TTF)
- In the combination arm (vismodegib + temozolomide): to further evaluate the safety of
the combination.
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