Prevention of Dichloroacetate Toxicity

• Status: Completed
• Trial end date: 2005-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a study to determine the safety of dichloroacetate (DCA) with a low-tyrosine diet given with or without nitisinone (NTBC) in children with chronic lactic acidosis (CLA).

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Florida
• Treatments: Nitisinone

Criteria

Inclusion criteria:

• Biochemical or molecular genetic proof of a defect in mitochondrial enzyme of glucose metabolism or oxidative phosphorylation.

• Clinical history consistent with CLA (e.g., basal hyperlactatemia, stroke-like episodes, neuromuscular degeneration, and seizures).

• Ability to withstand an 8-hour fast (if 2 years old or younger) or a 12-hour fast without developing hypoglycemia (blood glucose greater than or equal to 50 mg/dL).

Exclusion criteria:

• Secondary lactic acidosis due to impaired oxygenation or circulation.

• Hyperlactatemia associated with proven biotinidase deficiency or with enzyme deficiencies of gluconeogenesis.

• Primary, defined organic acidurias other than lactic acidosis for which effective therapy is available (e.g., propionic aciduria).

• Primary disorders of amino acid metabolism.

• Primary disorders of fatty acid oxidation.

• Malabsorption syndromes associated with D-lactic acidosis.

• Renal insufficiency.

• Serum creatinine greater than 1.2 mg/g.

• Creatinine clearance less than or equal to 60 mL/min.

• Primary hepatic disease unrelated to chronic lactic acidosis.

• In patients with pyruvate dehydrogenase enzyme complex deficiency, an inability to maintain a diet greater than 50% calories from fat without biological and/or neurological deterioration.