Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis
sodium dichloroacetate
Acid-Base Imbalance+9
+ Acidosis
+ Metabolic Diseases
Treatment Study
Summary
Study start date: October 1, 1998
Actual date on which the first participant was enrolled.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
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.45 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 3 Months to 18 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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