This open-label study evaluated the safety and efficacy of TSC when dosed concomitantly with the standard of care (radiation therapy and temozolomide) for newly diagnosed glioblastoma in adults. All patients received TSC in the study. The objective of the study was to evaluate the effect of TSC on survival and tumor response in patients with GBM while establishing an acceptable patient risk profile.
The overall objectives of this Phase 1/2 clinical study in newly diagnosed GBM patients was to evaluate the safety and tolerability, efficacy, PK profile, PFS/time to disease progression, QoL, and overall survival in adults when TSC is added to the standard of care regimen of radiation therapy and temozolomide. All patients received TSC in this study. The primary objective of the Phase 1 portion of the study was to evaluate the safety (DLT rate) and to define the dosing regimen of TSC for the larger Phase 2 study. The primary clinical endpoint was overall survival at 24 months and patients will be followed for up to 3 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
59
TSC administered intravenously as a bolus injection prior to radiation therapy sessions during 6 weeks of radiotherapy.
St. Joseph's Medical Center Barrow Neurology Clinics
Phoenix, Arizona, United States
Dose Limiting Toxicities (DLTs)
Number of Participants in Phase 1 with Dose Limiting Toxicities (DLTs)
Time frame: During phase 1
Overall Survival
Participants in phase 2 (18 dose group, 6 weeks treatment with TSC) were monitored for up to 3 years (last follow-up - February 16, 2016). Overall Survival (OS) was defined as the length of time from the date of tumor resection surgery or definitive biopsy to the date of death. The OS analyses were performed using the Kaplan-Meier estimate method. The OS rates at 6, 12, 18 and 24 months were estimated. Median OS values were calculated; a corresponding 95% confidence interval for each median value was determined using a log rank analysis. The length of OS (in months) was calculated as follows: date of death or censored - date of surgery or definitive biopsy / 30.4375.
Time frame: 6, 12, 18, 24 months
Progression-Free Survival (PFS)
The PFS analyses were performed using the Kaplan-Meier estimate method. The PFS rates at 6, 12, 18 and 24 months were estimated. Median PFS values were calculated; a corresponding 95% confidence interval for each median value was determined using a log rank analysis. Time to disease progression (in months) was calculated as follows: date of event\* or censoring - date of surgery or definitive biopsy / 30.4375; \*event = first tumor progression or death.
Time frame: 6,12,18, 24 months
Number of Participants With Reduction in Tumor Size, According to Percentage of Tumor Reduction
The sum of the product of the diameters of the tumor (using recorded tumor diameter measurements made from brain MRI images) was used to express tumor size. Results were summarized for actual and percentage change from baseline. Individual subjects results were listed, including tumor volume and tumor response from independent reviewers. Investigator data were listed but not used in the analysis. Percent response (according to independent reviewer assessments) by percentage tumor reduction from tumor resection or definitive biopsy to the last MRI were summarized.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of Arkansas Winthrop P. Rockefeller Cancer Institute
Little Rock, Arkansas, United States
USC/Norris Comprehensive Cancer Center
Los Angeles, California, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
The George Washington University Medical Center
Washington D.C., District of Columbia, United States
University of Florida McKnight Brain Institute
Gainesville, Florida, United States
Emory University Winship Cancer Institute
Atlanta, Georgia, United States
Northwestern University Robert H. Lurie Comprehensive Cancer Center
Chicago, Illinois, United States
Rush University Medical Center
Chicago, Illinois, United States
University of Kentucky Markey Cancer Center
Lexington, Kentucky, United States
...and 8 more locations
Time frame: From Baseline to Week 110