Study of the Safety and Efficacy of Dichloroacetate (DCA) in Brain Tumors - Full Text View

Sponsor:	University of Florida
Collaborator:	National Center for Research Resources (NCRR)
Information provided by (Responsible Party):	University of Florida
ClinicalTrials.gov Identifier:	NCT01111097

Purpose

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.

Condition	Intervention	Phase
Brain Tumor	Drug: Dichloroacetate	Phase I

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Phase 1, Open-Label, Single-Arm, Clinical and Metabolomics Study of Dichloroacetate (DCA) in Adults With Recurrent Malignant Brain Tumors

Resource links provided by NLM:

MedlinePlus related topics: Brain Cancer Cancer

Drug Information available for: Dichloroacetate Sodium dichloroacetate

U.S. FDA Resources

Further study details as provided by University of Florida:

Primary Outcome Measures:

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.

Secondary Outcome Measures:

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.

Estimated Enrollment:	15
Study Start Date:	April 2010
Estimated Study Completion Date:	April 2012
Estimated Primary Completion Date:	April 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: Cohort 1 Dosing of oral DCA is 8 mg/kg twice a day	Drug: Dichloroacetate DCA 50 mg/ml - Dose determined based on assigned cohort Other Name: DCA
Active Comparator: Cohort 2 Dosing of oral DCA is 12.5 mg/kg twice a day	Drug: Dichloroacetate DCA 50 mg/ml - Dose determined based on assigned cohort Other Name: DCA
Active Comparator: Cohort 3 Dosing of oral DCA is 5 mg/kg twice a day	Drug: Dichloroacetate DCA 50 mg/ml - Dose determined based on assigned cohort Other Name: DCA

Detailed 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.

Eligibility

Ages Eligible for Study:	21 Years to 90 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01111097

Locations

United States, Florida
University of Florida
Gainesville, Florida, United States, 32610

Sponsors and Collaborators

University of Florida

National Center for Research Resources (NCRR)

Investigators

Principal Investigator:	Erin M. Dunbar, MD	University of Florida
Study Chair:	Peter W. Stacpoole, PhD, MD	University of Florida

More Information

No publications provided

Responsible Party:	University of Florida
ClinicalTrials.gov Identifier:	NCT01111097 History of Changes
Other Study ID Numbers:	99-2010, 795
Study First Received:	April 23, 2010
Last Updated:	October 26, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by University of Florida:

Brain tumors and DCA
Brain tumor, recurrent

Additional relevant MeSH terms:

Brain Neoplasms
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site

Neoplasms
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on February 09, 2012