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

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT01111097
First received: April 23, 2010
Last updated: August 22, 2014
Last verified: August 2014

April 23, 2010
August 22, 2014
April 2010
March 2014   (final data collection date for primary outcome measure)
Determine the safety and tolerability of DCA in RMBTs. [ Time Frame: Within 28 days of starting DCA +/- 3 days ] [ Designated as safety issue: Yes ]
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.
Same as current
Complete list of historical versions of study NCT01111097 on ClinicalTrials.gov Archive Site
Conduct an exploratory investigation of the metabolites of patients with RMBTs and the effects of DCA thereon. [ Time Frame: One year ] [ Designated as safety issue: No ]
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.
Conduct an exploratory investigation of the metabolome of patients with RMBTs and the effects of DCA thereon. [ Time Frame: One year ] [ Designated as safety issue: No ]
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.
Not Provided
Not Provided
 
Study of the Safety and Efficacy of Dichloroacetate (DCA) in Glioblastoma and Other Recurrent Brain Tumors
Phase 1, Open-Label, Single-Arm, Clinical and Metabolomics Study of Dichloroacetate (DCA) in Adults With Recurrent Malignant 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.

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.

Interventional
Phase 1
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Brain Tumor
  • Glioblastoma
Drug: Dichloroacetate
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 Name: DCA
  • Active Comparator: Cohort 1
    Subjects are given a dose of Dichloroacetate 4mg/kg twice a day for 30 days
    Intervention: Drug: Dichloroacetate
  • Active Comparator: Cohort 2
    Subjects are given a dose of Dichloroacetate 12.5mg/kg twice a day for 30 days
    Intervention: Drug: Dichloroacetate
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
15
March 2014
March 2014   (final data collection date for primary outcome measure)

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.
Both
21 Years to 90 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01111097
99-2010, CTSI
Yes
University of Florida
University of Florida
National Center for Research Resources (NCRR)
Principal Investigator: Erin M. Dunbar, MD University of Florida
Study Chair: Peter W. Stacpoole, PhD, MD University of Florida
University of Florida
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP