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

Lactic Acidosis Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004493
• Other study ID #: 199/14274
• Secondary ID: UCSD-FDR001481
• Status: Completed
• Phase: Phase 2
• First received: October 18, 1999
• Last updated: March 24, 2015
• Start date: September 1998
• Est. completion date: September 2003

Study information

• Verified date: May 2000
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 75
• Est. completion date: September 2003
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Months and older

Eligibility

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

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Acidosis
• Acidosis, Lactic
• Lactic Acidosis

Intervention

Drug:
• sodium dichloroacetate

Locations

Country	Name	City	State
United States	University of California San Diego School of Medicine	La Jolla	California

Sponsors (1)

Lead Sponsor	Collaborator
University of California, San Diego

Country where clinical trial is conducted

United States,