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

This study has been completed.
Sponsor:
Collaborator:
University of California, San Diego
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004493
First received: October 18, 1999
Last updated: July 9, 2008
Last verified: May 2000
History of Changes
  Purpose

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
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.

  Eligibility

Ages Eligible for Study:   3 Months and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004493

Locations
United States, California
University of California San Diego School of Medicine
La Jolla, California, United States, 92093-0652
Sponsors and Collaborators
FDA Office of Orphan Products Development
University of California, San Diego
Investigators
Study Chair: Bruce Barshop University of California, San Diego
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004493     History of Changes
Other Study ID Numbers: 199/14274, UCSD-FDR001481
Study First Received: October 18, 1999
Last Updated: July 9, 2008
Health Authority: United States: Federal Government

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

Additional relevant MeSH terms:
Acidosis
Acidosis, Lactic
Acid-Base Imbalance
Metabolic Diseases

ClinicalTrials.gov processed this record on May 21, 2013