Study of the Safety and Efficacy of Dichloroacetate (DCA) in Glioblastoma and Other Recurrent Brain Tumors

University of Florida

Status and phase: Completed; Phase 1

Conditions: Glioblastoma; Brain Tumor

Treatments: Drug: Dichloroacetate

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT01111097

CTSI (Other Identifier)

99-2010

UL1TR000064 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

The purpose of this study is to evaluate the safety and tolerability of oral Dichloroacetate (DCA) in the treatment of recurrent malignant brain tumors (RMBTs). 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. Otherwise, there are no limitations to the number of prior recurrences. There are no limitations to the number or types of prior therapies.

Full description

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.

Enrollment: 15 patients

Sex: All

Ages: 21 to 90 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subject must be able to consent for self. Subject must have either:

  1. a brain metastasis or
  2. a WHO III-IV glioma that has recurred at least once. Females of child bearing age must have (-) pregnancy test.

• Females of child bearing age must use birth control while in study.

• Adequate organ function as determined by laboratory testing.

• Absence of peripheral neuropathy of moderate or greater severity (physician determined).

• Greater than 4 weeks time from previous anti-neoplastic (anti-cancer) therapy.

• Subject must have a Karnofsky Performance Status (KPS) of greater than or equal to 60.

• Subject must have an ECOG performance status of less than or equal to 2.

• There are no limitations to the number of prior recurrences.

• There are no limitations to the number or types of prior therapies.

Exclusion criteria

• Medical contraindication for magnetic resonance imaging (MRI)testing.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

15 participants in 2 patient groups

Cohort 1

Active Comparator group

Description:

Subjects are given a dose of Dichloroacetate 4mg/kg twice a day for 30 days

Treatment:

Drug: Dichloroacetate

Cohort 2

Active Comparator group

Description:

Subjects are given a dose of Dichloroacetate 12.5mg/kg twice a day for 30 days

Treatment:

Drug: Dichloroacetate