Dichloroacetate for Type 1 Diabetes

Overseen By Alice Hahn

Age: 18 - 65

Sex: Any

Trial Phase: Phase < 1

Sponsor: Yale University

Disqualifiers: Age, Weight, BMI, Retinopathy, others

Compensation: Varies

Number of Visits: 2

Duration: 1 day per treatment arm

16 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how dichloroacetate affects brain function when blood sugar drops too low in people with Type 1 Diabetes. The trial aims to determine if dichloroacetate can help manage hypoglycemia (low blood sugar) episodes, which can be dangerous if not treated quickly. Participants will receive either dichloroacetate or a placebo (a harmless pill with no effect) to compare results. Individuals with Type 1 Diabetes who have a history of severe low blood sugar episodes, especially those requiring assistance during these episodes, might be suitable for this trial. As an Early Phase 1 trial, this research focuses on understanding how dichloroacetate works in people, offering participants a chance to contribute to groundbreaking diabetes treatment research.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that dichloroacetate is likely to be safe for humans?

Research shows that dichloroacetate (DCA) has been safely used in humans for over 40 years. People with a rare metabolic disorder have received it without serious side effects. In some studies, participants reported mild sleepiness, but nothing severe.

Other studies tested DCA in people with diabetes for a week and found no major safety concerns. The long history of DCA use and various studies suggest it is generally well-tolerated. However, since this trial is in an early stage, limited information exists on its safety specifically for this condition. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Type 1 Diabetes?

Most treatments for Type 1 Diabetes, like insulin therapy, focus on managing blood sugar levels. But Dichloroacetate works differently by targeting the underlying metabolism. Researchers are excited because it has the potential to improve how the body uses glucose, which could reduce the reliance on frequent insulin injections. This unique mechanism could offer a new way to manage Type 1 Diabetes, making life easier for patients.

What evidence suggests that dichloroacetate might be an effective treatment for type 1 diabetes?

Research has shown that dichloroacetate (DCA) may benefit people with diabetes. In clinical trials, DCA lowered blood sugar levels in diabetic patients after just 6 to 7 days of oral administration. It has been safely used in humans for a long time, particularly for a rare metabolic disorder. Animal studies also suggest that DCA can improve diabetes-related issues, such as cataracts and nerve problems. This trial will evaluate DCA's potential to help manage Type 1 Diabetes symptoms, especially during low blood sugar episodes. Participants in this trial will receive either DCA or a placebo. ^{1 2 3 4 6}

Who Is on the Research Team?

Raimund Herzog, MD

Principal Investigator

Yale University

Are You a Good Fit for This Trial?

This trial is for people with Type 1 Diabetes who've had severe low blood sugar episodes or don't feel when their blood sugar gets too low. They should have needed help due to low blood sugar or had multiple very low readings on a continuous glucose monitor before the study.

Inclusion Criteria

I have type 1 diabetes and my blood sugar dropped below 54mg/dl twice last week.

I have type 1 diabetes with a history of severe low blood sugar or unawareness of low blood sugar.

T1DM subjects with a history of severe hypoglycemia with a blood glucose <54 mg/dL, requiring assistance of another person for recovery

Exclusion Criteria

My BMI is over 30.

I carry a gene variant linked to higher risk of drug toxicity.

I have an eye condition that has not been treated.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive dichloroacetate or placebo in a crossover design to assess brain glucose metabolism under clamped hypoglycemia

1 day per treatment arm

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Dichloroacetate

Trial Overview

The study tests Dichloroacetate's effect on brain function during controlled periods of low blood sugar in those with Type 1 Diabetes. It's a pilot study where participants are randomly given either the drug or a placebo without knowing which one they receive, and then switch.

How Is the Trial Designed?

Group I: Dichloroacetate Active Control 1 Intervention

oral solution of dichloroacetate with flavoring agent

Group II: Placebo Placebo Group 1 Intervention

oral solution with flavoring agent, no active drug.

Who Is Running the Clinical Trial?

Yale University

Lead Sponsor

Trials

1,963

Recruited

3,046,000+

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12419407/

Advances in the therapeutic applications of dichloroacetate as ...

Animal studies showed that DCA significantly reduced lens clouding in diabetic rats and dose-dependently improved diabetic cataract formation ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11756200/

Investigation of the protective effects of dichloroacetic acid ...

1b; Table 1). Diabetic rats administered 5 mg/kg of DCA displayed an improvement in CMAP, with values reaching 8.3 ± 0.5 mV in comparison with ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05317455?term=dichloroacetate&rank=7

Regulation of Brain Glucose Metabolism in Type 1 Diabetes

Dichloroacetate has a long safety record of administration to humans with a rare metabolic disorder for over 40 years. It has been given orally to patients with ...

4.

journals.lww.com

journals.lww.com/md-journal/fulltext/2025/09050/advances_in_the_therapeutic_applications_of.46.aspx

Advances in the therapeutic applications of dichloroacetate ...

In clinical trials, oral administration of DCA (3–4 g) in diabetic patients for 6 to 7 days significantly reduced fasting blood glucose levels, ...

5.

withpower.com

withpower.com/trial/dichloroacetate-for-type-1-diabetes-c4b41

Dichloroacetate for Type 1 Diabetes

Trial Overview The study tests Dichloroacetate's effect on brain function during controlled periods of low blood sugar in those with Type 1 Diabetes. It's a ...

6.

diabetesjournals.org

diabetesjournals.org/diabetes/article/68/Supplement_1/7-LB/56299/7-LB-Dichloroacetate-Improves-Cognitive-Function

7-LB: Dichloroacetate Improves Cognitive Function during ...

These results suggest that inhibition of PDH flux by enhanced PDK activity significantly contributes to cognitive impairment under hypoglycemia.