Clinical Trial: Trial Comparing the Effects of Intermittent Vismodegib vs. PDT in Patients With Multiple Basal Cell Carcinomas
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase II Randomized, Open Label Trial Comparing the Effects of Intermittent Vismodegib Versus PDT on the Maintenance of Benefit Following 7 Months of Continuous Vismodegib Treatment in Patients With
Brief Summary: The purpose of this study is to evaluate and compare the safety and efficacy of intermittent vismodegib and of Photodynamic Therapy (PDT).
Detailed Summary: This is a Phase II, 28 month, randomized, two arm multicenter clinical study design. During the initial 7 months of the study, all 24 subjects will receive vismodegib, 150mg/day. They then will be randomized in a 1:1 ratio to receive intermittent vismodegib, 150 mg/day, during months 10-13, 16-19, and 22-25 or to receive treatment with PDT at month 10 and at three month intervals thereafter. The safety and efficacy of intermittent vismodegib and of PDT will be assessed at the time of the subjects' visits to the Study Center and at the time of telephone contacts. A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months. The DSMB review will focus on adverse events and efficacy results. Subjects will be monitored for the presence of surrogate endpoint biomarkers (SEBs) at each Study visit.
Sponsor: Children's Hospital & Research Center Oakland
Current Primary Outcome: Time to recurrence to baseline SEB burden following 7 months of continuous vismodegib treatment. [ Time Frame: A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months. ]
Primary:
i. To compare the efficacy of (i) intermittent vismodegib vs. (ii) the efficacy of photodynamic therapy (PDT) in preventing the return of the burden of surgically eligible BCCs (SEBs) to baseline level following 7 months of continuous vismodegib therapy.
ii. To compare the cumulative diameter (burden) of SEBs in patients treated intermittently with vismodegib vs. with photodynamic therapy (PDT).
Original Primary Outcome: Same as current
Current Secondary Outcome: The cumulative diameter (burden) of SEBs in patients treated intermittently with vismodegib vs PDT during months 8-28 maintenance period. [ Time Frame: A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months. ]
i. To assess the safety of intermittent vismodegib in patients with multiple BCCs (BCNS and non-BCNS) during months 8-28.
ii. To assess resistance of SEBs to treatments in patients with multiple BCCs (BCNS and non-BCNS) treated intermittently during months 8-28.
iii. To assess the degree of reduction of SEBs after 7 months of continuous daily vismodegib therapy.
iv. To conduct an exploratory evaluation in non-BCNS patients with multiple BCCs (high burden of disease) of the efficacy and tolerability of intermittent vismodegib vs PDT
Original Secondary Outcome: Same as current
Information By: Children's Hospital & Research Center Oakland
Dates:
Date Received: March 14, 2012
Date Started: April 2012
Date Completion:
Last Updated: January 13, 2016
Last Verified: January 2016
