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NCT Number: NCT01111097

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

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.

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Key information

Conditions

Brain Tumor Astrocytoma Brain Diseases Brain Neoplasms Central Nervous System Diseases Central Nervous System Neoplasms Glioblastoma Glioma Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Nervous System Diseases Nervous System Neoplasms Neuroectodermal Tumors

Age range

21 year–90 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Florida

Gainesville, Florida, 32610, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subject must be able to consent for self. Subject must have either:
  • a brain metastasis or
  • 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.

Treatment and study plan

Dichloroacetate

Drug

Subjects after passing the inclusion criteria are given a dose of dichloroacetate 4mg/kg bid for thirty days. While in the clinical research center they participate in a breath test where they exhale through a straw into a glass tube. This will measure CO2. They are monitored every two weeks for side effects and return to the clinical research center for evaluation in thirty days. They undergo another breath test and if all health parameters are within normal limits they are given a month's supply of dichloroacetate. The cycles continue unless a serious adverse event occurs or the PI judges the side effects preclude another 30 days of medication

Other names: DCA

Primary outcomes

  1. Determine the safety and tolerability of DCA in RMBTs.

    Time frame: Within 28 days of starting DCA +/- 3 days

    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.

Secondary outcomes

  1. Conduct an exploratory investigation of the metabolites of patients with RMBTs and the effects of DCA thereon.

    Time frame: One year

    We postulate that the metabolism of RMBTs and the effects of DCA thereon will help investigators understand RMBTs, how DCA works on them, and how to design future treatment studies.

Sponsors and collaborators

Lead sponsor

University of Florida

Other

Collaborators

  • National Institutes of Health (NIH)

Registry information

Official study title

Phase 1, Open-Label, Single-Arm, Clinical and Metabolomics Study of Dichloroacetate (DCA) in Adults With Recurrent Malignant Brain Tumors

Important dates

Study start
2010
Primary completion
2014
Study completion
2014
First posted
Apr 27, 2010
Registry last updated
Sep 22, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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