Open-label, Single Arm, Safety and Tolerability Dose Escalation Study of VAL-083 in Patients With Recurrent Malignant Glioma or Progressive Secondary Brain Tumor
Recurrent glial tumors of the brain continue to be one of the most challenging malignancies
to treat. Median survival for patients with recurrent disease is approximately 6 months for
glioblastoma multiforme. Bevacizumab is used for treatment of recurrent disease; however
patients who fail bevacizumab do not have many treatment options.
Metastases to the brain are the most common intracranial tumors in adults and occur ten
times more frequently than primary brain tumors. It is estimated that 8 - 10% of cancer
patients may develop symptomatic metastatic tumors in the brain. Systemic therapy is rarely
used for primary treatment of brain metastases because many tumors that metastasize to the
brain are not chemosensitive or have been already heavily pretreated with potentially
effective agents, and poor penetration through the blood brain barrier is an additional
Dianhydrogalactitol (DAG) rapidly penetrates both the cerebrospinal fluid (CSF) and the
blood-brain barrier and accumulates in brain tissue. Clinical study of DAG in patients with
GBM or with progressive secondary brain tumors is warranted.
This study will utilize a standard 3 + 3 dose escalation design, until the MTD or the
maximum specified dose has been reached. In Phase 2, additional patients with GBM or
progressive secondary brain tumor will be treated at the MTD (or other selected optimum
Phase 2 dose) to measure tumor responses to treatment.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Determination of maximum tolerated dose (MTD)
The determination of MTD will be based on analysis of tolerance data from the first cycle of therapy in each dose group.
Study Day 35
Howard A Burris, M.D.
Sarah Cannon Research Institute; Nashville, TN 37203, USA
United States: Food and Drug Administration
|Florida Cancer Specialists||Fort Myers, Florida 33901|
|Sarah Cannon Research Institute||Nashville, Tennessee 37203|