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

Trial Summary

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 dichloroacetate generally safe for humans?

Dichloroacetate has been used in studies for various conditions, and while some patients experienced mild sedation, no serious side effects were noted in short-term use. However, it can cause reversible peripheral neuropathy (nerve damage) that might be related to thiamine deficiency, and this can be managed with thiamine supplements. ^{1 2 3 4 5}

What is the purpose of this trial?

This is a prospective randomized placebo-controlled double-blind crossover pilot study determining the effect of dichloroacetate on brain function under clamped hypoglycemia in T1DM.

Research Team

Raimund Herzog, MD

Principal Investigator

Yale University

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

1. United States pubmed.ncbi.nlm.nih.gov

Metabolic effects of dichloroacetate in patients with diabetes mellitus and hyperlipoproteinemia. [2013]

2. United States pubmed.ncbi.nlm.nih.gov

The pharmacology of dichloroacetate. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Evaluation of long-term treatment of children with congenital lactic acidosis with dichloroacetate. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Dichloroacetate. [2019]

5. England pubmed.ncbi.nlm.nih.gov

Pharmacokinetics of dichloroacetate in adult patients with lactic acidosis. [2019]