NCT00004493

Brief Summary

OBJECTIVES: I. Determine the pharmacokinetics of sodium dichloroacetate (DCA) in patients with congenital lactic acidemia. II. Determine the efficacy of DCA in decreasing the frequency and/or severity of acute episodes of acidotic illness, improving linear growth, improving neurological or developmental function, or slowing neurological or developmental deterioration in these patients.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
75

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Sep 1998

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 1998

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
3.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2003

Completed
Last Updated

March 25, 2015

Status Verified

May 1, 2000

First QC Date

October 18, 1999

Last Update Submit

March 24, 2015

Conditions

Lactic Acidosis

Keywords

inborn errors of metabolismlactic acidosisrare disease

Interventions

sodium dichloroacetateDRUG

Eligibility Criteria

Age3 Months+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Diagnosis of stable, persistent lactic acidemia Venous lactate at least 3 mM under basal conditions defined as: At least 4 hours postprandial No concurrent illness OR * Diagnosis of cerebral lactic acidemia with elevated lactic acid in CSF but not in the blood * No organic acidemias or defective gluconeogenesis --Patient Characteristics-- * Hematopoietic: Hemoglobin at least 7 mg/dL * Hepatic: Bilirubin no greater than 3 times upper limit of normal (ULN) AST, ALT, or GGT no greater than 10 times ULN * Renal: Creatinine no greater than 2 mg/dL * Cardiovascular: Ejection fraction at least 25% * Other: No hypoglycemia (blood sugar less than 50 mg/dL at no greater than 12 hours fasting) No severe peripheral neuropathy interfering with normal activities of living

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

University of California San Diego School of Medicine

La Jolla, California, 92093-0652, United States

Location

MeSH Terms

Conditions

Acidosis, LacticMetabolism, Inborn ErrorsRare Diseases

Interventions

Dichloroacetic Acid

Condition Hierarchy (Ancestors)

AcidosisAcid-Base ImbalanceMetabolic DiseasesNutritional and Metabolic DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

ChloroacetatesAcetatesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsHydrocarbons, ChlorinatedHydrocarbons, HalogenatedHydrocarbons

Study Officials

  • Bruce Barshop

    University of California, San Diego

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

September 1, 1998

Study Completion

September 1, 2003

Last Updated

March 25, 2015

Record last verified: 2000-05

Locations

US(1)