- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01111097
Study of the Safety and Efficacy of Dichloroacetate (DCA) in Glioblastoma and Other Recurrent Brain Tumors
September 18, 2015 updated by: University of Florida
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.
Study Overview
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.
Study Type
Interventional
Enrollment (Actual)
15
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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Florida
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Gainesville, Florida, United States, 32610
- University of Florida
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
19 years to 88 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: Cohort 1
Subjects are given a dose of Dichloroacetate 4mg/kg twice a day for 30 days
|
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:
|
Active Comparator: Cohort 2
Subjects are given a dose of Dichloroacetate 12.5mg/kg twice a day for 30 days
|
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:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
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Within 28 days of starting DCA +/- 3 days
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Conduct an exploratory investigation of the metabolites of patients with RMBTs and the effects of DCA thereon.
Time Frame: One year
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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.
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One year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Erin M. Dunbar, MD, University of Florida
- Study Chair: Peter W. Stacpoole, PhD, MD, University of Florida
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
April 1, 2010
Primary Completion (Actual)
March 1, 2014
Study Completion (Actual)
March 1, 2014
Study Registration Dates
First Submitted
April 23, 2010
First Submitted That Met QC Criteria
April 26, 2010
First Posted (Estimate)
April 27, 2010
Study Record Updates
Last Update Posted (Estimate)
September 22, 2015
Last Update Submitted That Met QC Criteria
September 18, 2015
Last Verified
September 1, 2015
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Neoplasms by Site
- Neoplasms, Glandular and Epithelial
- Astrocytoma
- Glioma
- Neoplasms, Neuroepithelial
- Neuroectodermal Tumors
- Neoplasms, Germ Cell and Embryonal
- Neoplasms, Nerve Tissue
- Central Nervous System Neoplasms
- Nervous System Neoplasms
- Glioblastoma
- Brain Neoplasms
Other Study ID Numbers
- 99-2010
- UL1TR000064 (U.S. NIH Grant/Contract)
- CTSI (Other Identifier: Ocala Dames)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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