The Sapphire study will be carried out in Europe, America and Asia. Approximately 70 hospital centers will participate. The Phase III study is designed as a randomized, active-controlled, open-label clinical trial with two treatment arms: trabedersen in a dose of 10 micromolar concentration will be compared to current standard therapy with either temozolomide or BCNU.
Clinical centers conducting the Sapphire study aim to enroll a total of approximately 130 adult patients with recurrent or refractory anaplastic astrocytoma. Trabedersen will be administered intratumorally via one single catheter using convection-enhanced delivery on an outpatient treatment basis. The treatment period lasts up to six months consisting of seven-day cycles every other week.
The primary efficacy endpoint is the survival rate at 24 months. Further efficacy endpoints include overall survival and time to death. The 14-month progression rate is the surrogate endpoint for an interim analysis.
The results of the previous randomized, active-controlled Phase IIb study show that the novel, targeted therapy holds much promise, said Antisense Pharma.
Karl-Hermann Schlingensiepen, CEO of Antisense Pharma, said: “We have taken trabedersen all the way from drug discovery to the pivotal Phase III clinical trial. The enrollment of the first patients in the Sapphire study is a key milestone in our drug development program for trabedersen.
“It is also in Phase I/II clinical development for advanced pancreatic carcinoma, malignant melanoma and colorectal carcinoma. Trabedersen with its unique mode of action can lead to a paradigm shift towards tackling malignant tumors at their roots while providing a better quality of life for patients.”