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

Tracking Information

First Received Date ^ICMJE

October 18, 1999

Last Updated Date

July 9, 2008

Start Date ^ICMJE

September 1998

Primary Completion Date

Current Primary Outcome Measures ^ICMJE

Original Primary Outcome Measures ^ICMJE

Change History

Complete list of historical versions of study NCT00004493 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

Brief Summary

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.

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.

Study Phase

Phase II

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment

Condition ^ICMJE

Lactic Acidosis

Intervention ^ICMJE

Drug: sodium dichloroacetate

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Estimated Enrollment ^ICMJE

Completion Date

Primary Completion Date

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

3 Months and older

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT ID ^ICMJE

NCT00004493

Responsible Party

Study ID Numbers ^ICMJE

199/14274, UCSD-FDR001481

Study Sponsor ^ICMJE

FDA Office of Orphan Products Development

Collaborators ^ICMJE

University of California, San Diego

Investigators ^ICMJE

Study Chair:

Bruce Barshop

University of California, San Diego

Information Provided By

FDA Office of Orphan Products Development

Verification Date

May 2000

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP