NCT01601184

Study of Vismodegib in Combination With Temozolomide Versus Temozolomide Alone in Patients With Medulloblastomas With an Activation of the Sonic Hedgehog Pathway

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Centre Leon Berard · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Histologically Confirmed Medulloblastoma
• Activation of the Sonic Hedgehog (SHH) Pathway

Interventions

Type	Name	Description
DRUG	vismodegib	Hedgehog pathway antagonist Dosage: 150 mg orally with or without food at the same time every day
DRUG	Temozolomide	alkylating agent Dosage: Dose in Cycle 1 is 150 mg/m2 orally once daily for 5 days followed by 23 days without treatment. At the start of Cycle 2, the dose is escalated to 200mg/m2 orally once daily for 5 days

Timeline

• Start date: 2012-06-01
• Primary completion: 2017-09-01
• Completion: 2017-10-01
• First posted: 2012-05-17
• Last updated: 2019-05-21

Locations

15 sites across 4 countries: France, Italy, Switzerland, United Kingdom