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

This study has been completed.
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: May 2000

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.

Condition Intervention Phase
Lactic Acidosis Drug: sodium dichloroacetate Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 75
Study Start Date: September 1998
Estimated Study Completion Date: September 2003
Detailed Description:

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


Ages Eligible for Study:   3 Months and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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


  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00004493

United States, California
University of California San Diego School of Medicine
La Jolla, California, United States, 92093-0652
Sponsors and Collaborators
University of California, San Diego
Study Chair: Bruce Barshop University of California, San Diego
  More Information Identifier: NCT00004493     History of Changes
Other Study ID Numbers: 199/14274
Study First Received: October 18, 1999
Last Updated: March 24, 2015

Keywords provided by FDA Office of Orphan Products Development:
inborn errors of metabolism
lactic acidosis
rare disease

Additional relevant MeSH terms:
Acidosis, Lactic
Acid-Base Imbalance
Metabolic Diseases processed this record on August 22, 2017