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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
  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-Blind
Primary Purpose: Treatment

Resource links provided by NLM:


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

Estimated Enrollment: 45
Study Start Date: October 1998
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.

  Eligibility

Ages Eligible for Study:   3 Months to 18 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004490

Sponsors and Collaborators
University of Florida
Investigators
Study Chair: Peter W. Stacpoole University of Florida
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004490     History of Changes
Other Study ID Numbers: 199/14271, UF-G-FDR001500, UF-G-183-92
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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

ClinicalTrials.gov processed this record on November 20, 2014