Phase 1, Open-Label, Single-Arm, Clinical and Metabolomics Study of Dichloroacetate (DCA) in Adults With Recurrent Malignant Brain Tumors
Malignant brain tumors are defined as any World Health Organization grade III-IV glioma and
any solid tumor metastasis (spread) to the brain. Recurrent malignant brain tumors (RMBTs)
are defined as either: 1) malignant tumors, originating in the brain, that have recurred at
least once or 2) malignant tumors originating elsewhere in the body that have spread to the
brain at least once. They share an increasing incidence, clinical and radiographic
characteristics, lack of effective therapies, tendency to recur, and poor outcome.
Importantly, recurrent malignant brain tumor's shared characteristics may be usefully
exploited by an emerging class of biologic agents called metabolic modulators of which
Dichloroacetate (DCA) is the drug in the class most thoroughly investigated clinically.
DCA's mechanism of action and tolerability have been extensively demonstrated in the
treatment of chronic metabolic disorders. Furthermore, the preciseness of DCA's mechanism
of action appears to target abnormal tumor cell metabolism.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Determine the safety and tolerability of DCA in RMBTs.
Oral DCA will be administered until intolerance, toxicity, radiographic progression, or death. Safety and tolerance will be assessed by reviewing available standardized clinical, radiographic, and quality of life (QOL) criteria. The safety and tolerance will also be assessed by reviewing available plasma, urine, and brain tumor tissue for metabolites of the tumor and the effects of DCA thereon.
Within 28 days of starting DCA +/- 3 days
Erin M. Dunbar, MD
University of Florida
United States: Food and Drug Administration
|University of Florida||Gainesville, Florida 32610-0277|