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

Primary Purpose

Lactic Acidosis

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
sodium dichloroacetate
Sponsored by
University of Florida
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lactic Acidosis focused on measuring disease-related problem/condition, inborn errors of metabolism, lactic acidosis, quality of life, rare disease

Eligibility Criteria

3 Months - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    October 18, 1999
    Last Updated
    March 24, 2015
    Sponsor
    University of Florida
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004490
    Brief Title
    Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2000
    Overall Recruitment Status
    Completed
    Study Start Date
    October 1998 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    September 2002 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    University of Florida

    4. Oversight

    5. Study Description

    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

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Lactic Acidosis
    Keywords
    disease-related problem/condition, inborn errors of metabolism, lactic acidosis, quality of life, rare disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Crossover Assignment
    Masking
    Double
    Allocation
    Randomized
    Enrollment
    45 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    sodium dichloroacetate

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    3 Months
    Maximum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Peter W. Stacpoole
    Organizational Affiliation
    University of Florida
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

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

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