Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia - Full Text View

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

This study has been completed.

Sponsors and Collaborators:	FDA Office of Orphan Products Development University of California, San Diego
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004493

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 II

Drug Information available for:

Dichloroacetate Sodium dichloroacetate

U.S. FDA Resources

Study Type:	Interventional
Study Design:	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

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

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

Keywords provided by FDA Office of Orphan Products Development:

inborn errors of metabolism

lactic acidosis

rare disease

Study placed in the following topic categories:

Metabolism, Inborn Errors

Metabolic Diseases

Acidosis, Lactic

Rare Diseases

Metabolic disorder

Acidosis

Additional relevant MeSH terms:

Acid-Base Imbalance

ClinicalTrials.gov processed this record on November 30, 2008