Could a common metabolic drug help fight brain cancer?
NCT ID NCT05120284
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This Phase 2 trial tests whether dichloroacetate (DCA), a drug that affects cell energy use, can alter tumor metabolism in people with recurrent glioblastoma. Forty patients scheduled for surgery will either receive DCA or no drug for one week before their operation. Researchers will then compare tumor tissue to see if DCA changes key proteins linked to cancer growth.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- dichloroacetate (DCA)
- What this could lead to
- If successful, this trial could show that DCA changes tumor metabolism in a way that might lead to new treatment options for glioblastoma.
- What could go wrong
- This is a small, early-phase study focused on biological markers, not survival or tumor shrinkage. The drug may not show meaningful clinical benefit, and side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 40 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2022
- Expected to finish
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Aug 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 80 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Study subjects will be male and female adults, aged 18 through 80 years, previously diagnosed with a GBM who have experienced tumor recurrence as determined by neuroimaging and some degree of symptomatology (e.g., headache, mental status change, seizure) and have clinically indicated tumor debulking surgery planned. * All subjects will have completed initial, standard- therapy with surgical debulking, followed by radiation and temozolomide (TMZ) and will, therefore, be considered treatment failures. Patients with truly unmethylated GBM do not require prior treatment with temozolomide (TMZ). * Patients will be recruited and studied at Johns Hopkins University, Johns Hopkins affiliated Sibley Memorial Hospital, and Wake Forest University. The DCA liquid formulation is on file with the FDA, is identical to that administered in our Phase I trial of brain tumor patients and can be given by mouth or feeding tube. Patients may retain whatever medications they are receiving for other conditions (e.g., hypertension, seizures), except patients requiring insulin or sulfonylurea therapy (see below). * The probability of adverse drug-drug interactions is extremely low, for the following reasons. First, DCA is the only pharmaceutical in clinical use that is metabolized by GSTZ1. Second, DCA is not known to be metabolized by any other drug metabolizing enzyme system, thus precluding competition with other agents for biotransformation. Third, the results of both open label and randomized controlled trials of orally or parenterally administered DCA in the treatment of children and/or adults have never shown evidence of adverse drug-drug interactions (34). Thus, from decades of clinical investigations of use of DCA in various acutely or chronically ill populations, there is nothing to suggest adverse drug-drug interactions should be anticipated in this trial. * Patients who are diabetic must have a screening hemoglobin A1c (Hgb A1c) level of at least 6.0. Exclusion Criteria: * Patients considered pre-terminal (life expectancy ≤ 2 months) * Those who are pregnant will be excluded. * DCA inhibits gluconeogenesis and lowers blood glucose levels in patients with type 2 diabetes. Therefore, in subjects who are receiving either insulin or a sulfonylurea, coadministration of DCA could lead to symptomatic hypoglycemia and those patients will be excluded from the trial. * DCA is dialyzable and its clearance diminishes in patients with end stage renal failure (GFR ≤ 30 ml/min); such patients will be excluded from participating. * DCA is metabolized by hepatic GSTZ1, so patients with severe liver insufficiency (total bilirubin \> 2.0 mg/dl or ALT or AST \> 3 x ULN) will be excluded. * Patients with Hgb A1c level less than 6.0 at screening
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, 21231, United States
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Wake Forest University
Winston-Salem, North Carolina, 27587, United States
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