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Completed

NCT Number: NCT00004493

Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia

OBJECTIVES: I. Determine the pharmacokinetics of sodium dichloroacetate (DCA) in patients with congenital lactic acidemia.

II. Determine the efficacy of DCA in decreasing the frequency and/or severity of acute episodes of acidotic illness, improving linear growth, improving neurological or developmental function, or slowing neurological or developmental deterioration in these patients.

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Key information

Conditions

Lactic Acidosis Acid-Base Imbalance Acidosis Acidosis, Lactic Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

3 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of California San Diego School of Medicine

La Jolla, California, 92093-0652, United States

About this study

PROTOCOL OUTLINE: This is a randomized, double blind, cross over study. Patients are randomized to start with one of two different doses of sodium dichloroacetate (DCA).

Patients receive one of two doses of oral DCA for 6 months, then switch to the alternate dose for 6 months. This course is repeated once.

Patients are followed for up to 2 years.

Completion date provided represents the completion date of the grant per OOPD records

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

  • Diagnosis of stable, persistent lactic acidemia Venous lactate at least 3 mM under basal conditions defined as: At least 4 hours postprandial No concurrent illness

OR

  • Diagnosis of cerebral lactic acidemia with elevated lactic acid in CSF but not in the blood
  • No organic acidemias or defective gluconeogenesis

--Patient Characteristics--

  • Hematopoietic: Hemoglobin at least 7 mg/dL
  • Hepatic: Bilirubin no greater than 3 times upper limit of normal (ULN) AST, ALT, or GGT no greater than 10 times ULN
  • Renal: Creatinine no greater than 2 mg/dL
  • Cardiovascular: Ejection fraction at least 25%
  • Other: No hypoglycemia (blood sugar less than 50 mg/dL at no greater than 12 hours fasting) No severe peripheral neuropathy interfering with normal activities of living

Treatment and study plan

sodium dichloroacetate

Drug

Sponsors and collaborators

Lead sponsor

University of California, San Diego

Other

Registry information

Important dates

Study start
1998
Study completion
2003
First posted
Oct 19, 1999
Registry last updated
Mar 25, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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