Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis

NCT ID: NCT00004490

Last Updated: 2015-03-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 45 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1998-10-31
• Study Completion Date: 2002-09-30

Brief Summary

OBJECTIVES:

I. Compare the safety of sodium dichloroacetate (DCA) vs placebo in children with congenital lactic acidosis.

II. Determine the quality of life of these patients.

III. Determine the pharmacokinetics and metabolic fate of DCA over the course of drug administration in these patients.

Detailed Description

PROTOCOL OUTLINE: This is a randomized, double blind, crossover study. Patients are stratified according to age (3 months to 2 years vs over 2 to 18 years).

All patients receive at least 12 months of sodium dichloroacetate (DCA) during a 2 year period of double blind, crossover evaluation of DCA and placebo by mouth.

Quality of life is assessed before treatment and periodically during treatment.

Completion date provided represents the completion date of the grant per OOPD records

Conditions

Lactic Acidosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Interventions

sodium dichloroacetate

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Diagnosis of congenital lactic acidosis (CLA) meeting the following criteria: Three basal venous lactates at least 2.5 mM, arterial lactates at least 2.0 mM, or CSF lactates at least 2.5 mM OR any combination of these, obtained over at least 1 month and within 6 months OR Increase in blood lactate at least 1.0 mM over basal following a carbohydrate meal challenge

AND

Enzymatic or molecular genetic proof of a defect of pyruvate dehydrogenase complex, one or more respiratory chain enzymes, or a Krebs cycle enzyme OR Over production of C14-lactate from C14-glucose by cultured skin fibroblasts

AND

Ability to withstand an 8 hour (if 2 years and under) or 12 hour (if over 2 years) fast without developing hypoglycemia (blood glucose less than 50 mg/dL)

No secondary lactic acidosis due to impaired oxygenation or circulation

No hyperlactatemia associated with proven biotinidase deficiency (biotin responsive CLA) or with enzyme deficiencies of gluconeogenesis

No primary, defined organic acidurias other than lactic acidosis, for which effective therapy is available (e.g., propionic aciduria)

No primary disorders of amino acid metabolism or fatty acid oxidation

No malabsorption syndromes associated with D-lactic acidosis

--Prior/Concurrent Therapy--

No chronic dialysis

--Patient Characteristics--

Hepatic: No primary hepatic disease unrelated to CLA

Renal: Creatinine less than 1.2 mg/dL OR Creatinine clearance at least 60 mL/min

Other: No concurrent infection or fever

• Minimum Eligible Age: 3 Months
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Florida

OTHER

Sponsor Role: lead

Principal Investigators

Peter W. Stacpoole

Role: STUDY_CHAIR

University of Florida

Other Identifiers

UF-G-FDR001500

Identifier Type: -

Identifier Source: secondary_id

UF-G-183-92

Identifier Type: -

Identifier Source: secondary_id

199/14271

Identifier Type: -

Identifier Source: org_study_id