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

This study has been completed.

Sponsor:

ClinicalTrials.gov Identifier:

NCT00004490

First Posted: October 19, 1999

Last Update Posted: March 25, 2015

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

Information provided by:

FDA Office of Orphan Products Development

Purpose

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.

Condition	Intervention	Phase
Lactic Acidosis	Drug: sodium dichloroacetate	Phase 3


Study Type:	Interventional
Study Design:	Allocation: Randomized Intervention Model: Crossover Assignment Masking: Double Primary Purpose: Treatment

Further study details as provided by FDA Office of Orphan Products Development:


Estimated Enrollment:	45
Study Start Date:	October 1998
Estimated Study Completion Date:	September 2002

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

Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:	3 Months to 18 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004490

Sponsors and Collaborators

University of Florida

Investigators


Study Chair:	Peter W. Stacpoole	University of Florida

More Information


ClinicalTrials.gov Identifier:	NCT00004490 History of Changes
Other Study ID Numbers:	199/14271 UF-G-FDR001500 UF-G-183-92
First Submitted:	October 18, 1999
First Posted:	October 19, 1999
Last Update Posted:	March 25, 2015
Last Verified:	April 2000

Keywords provided by FDA Office of Orphan Products Development:


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

Additional relevant MeSH terms:


Acidosis Acidosis, Lactic Acid-Base Imbalance Metabolic Diseases

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