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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2000 by FDA Office of Orphan Products Development.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
University of Florida
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004490
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: April 2000

October 18, 1999
June 23, 2005
October 1998
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Complete list of historical versions of study NCT00004490 on ClinicalTrials.gov Archive Site
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Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis
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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.

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.

Interventional
Phase 3
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Lactic Acidosis
Drug: sodium dichloroacetate
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
45
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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

Both
3 Months to 18 Years
No
Contact information is only displayed when the study is recruiting subjects
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NCT00004490
199/14271, UF-G-FDR001500, UF-G-183-92
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FDA Office of Orphan Products Development
University of Florida
Study Chair: Peter W. Stacpoole University of Florida
FDA Office of Orphan Products Development
April 2000

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP