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

NCT00004490 | Completed Phase 3

• 3 other identifiers: 199/14271UF-G-FDR001500UF-G-183-92
• Study Type: interventional
• Target: 45
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Lactic Acidosis

Keywords

disease-related problem/condition; inborn errors of metabolism; lactic acidosis; quality of life; rare disease

Interventions

sodium dichloroacetate DRUG

Eligibility Criteria

• Age: 3 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• University of Florida: lead

MeSH Terms

Conditions

Acidosis, Lactic; Metabolism, Inborn Errors; Rare Diseases

Interventions

Dichloroacetic Acid

Condition Hierarchy (Ancestors)

Acidosis; Acid-Base Imbalance; Metabolic Diseases; Nutritional and Metabolic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Chloroacetates; Acetates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Hydrocarbons, Chlorinated; Hydrocarbons, Halogenated; Hydrocarbons

Study Officials

• Peter W. Stacpoole

  University of Florida

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: October 1, 1998
• Study Completion: September 1, 2002
• Last Updated: March 25, 2015

Record last verified: 2000-04