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

NCT01111097 | Completed Phase 1 DMC

• 3 other identifiers: 99-2010CTSIUL1TR000064
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Brain Tumor; Glioblastoma

Keywords

Brain tumors, Glioblastoma and DCA; Brain tumor,Glioblastoma recurrent

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (1)

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

  One year

Study Arms (2)

Cohort 1

ACTIVE COMPARATOR

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

Drug: Dichloroacetate

Cohort 2

ACTIVE COMPARATOR

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

Drug: Dichloroacetate

Interventions

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

Also known as: DCA

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 21 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• University of Florida: lead
• National Institutes of Health (NIH): collaborator

Study Sites (1)

University of Florida

Gainesville, Florida, 32610, United States

Location

Related Publications (1)

• Dunbar EM, Coats BS, Shroads AL, Langaee T, Lew A, Forder JR, Shuster JJ, Wagner DA, Stacpoole PW. Phase 1 trial of dichloroacetate (DCA) in adults with recurrent malignant brain tumors. Invest New Drugs. 2014 Jun;32(3):452-64. doi: 10.1007/s10637-013-0047-4. Epub 2013 Dec 3.

  PMID: 24297161 RESULT

MeSH Terms

Conditions

Brain Neoplasms; Glioblastoma

Interventions

Dichloroacetic Acid

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Chloroacetates; Acetates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Hydrocarbons, Chlorinated; Hydrocarbons, Halogenated; Hydrocarbons

Study Officials

• Erin M. Dunbar, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

• Peter W. Stacpoole, PhD, MD

  University of Florida

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 23, 2010
• First Posted: April 27, 2010
• Study Start: April 1, 2010
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: September 22, 2015

Record last verified: 2015-09

Locations

US (1)