NCT00031161

Brief Summary

This is a study to determine the safety of dichloroacetate (DCA) with a low-tyrosine diet given with or without nitisinone (NTBC) in children with chronic lactic acidosis (CLA).

Trial Health

87
On Track

Trial Health Score

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

Enrollment
30

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Sep 2001

Longer than P75 for not_applicable

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, 2001

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

February 26, 2002

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 27, 2002

Completed
3.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2005

Completed
Last Updated

March 25, 2015

Status Verified

January 1, 2002

First QC Date

February 26, 2002

Last Update Submit

March 24, 2015

Conditions

Acidosis, LacticChronic Disease

Keywords

TyrosineDiet2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione4-Hydroxyphenylpyruvate DioxygenaseEnzyme Inhibitors

Interventions

Nitisinone (NTBC)DRUG
DichloroacetateDRUG
Low-tyrosin dietBEHAVIORAL

Eligibility Criteria

Age3 Months - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Biochemical or molecular genetic proof of a defect in mitochondrial enzyme of glucose metabolism or oxidative phosphorylation.
  • Clinical history consistent with CLA (e.g., basal hyperlactatemia, stroke-like episodes, neuromuscular degeneration, and seizures).
  • Ability to withstand an 8-hour fast (if 2 years old or younger) or a 12-hour fast without developing hypoglycemia (blood glucose greater than or equal to 50 mg/dL).

You may not qualify if:

  • Secondary lactic acidosis due to impaired oxygenation or circulation.
  • Hyperlactatemia associated with proven biotinidase deficiency or with enzyme deficiencies of gluconeogenesis.
  • Primary, defined organic acidurias other than lactic acidosis for which effective therapy is available (e.g., propionic aciduria).
  • Primary disorders of amino acid metabolism.
  • Primary disorders of fatty acid oxidation.
  • Malabsorption syndromes associated with D-lactic acidosis.
  • Renal insufficiency.
  • Serum creatinine greater than 1.2 mg/g.
  • Creatinine clearance less than or equal to 60 mL/min.
  • Primary hepatic disease unrelated to chronic lactic acidosis.
  • In patients with pyruvate dehydrogenase enzyme complex deficiency, an inability to maintain a diet greater than 50% calories from fat without biological and/or neurological deterioration.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Florida

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Acidosis, LacticChronic Disease

Interventions

nitisinoneDichloroacetic Acid

Condition Hierarchy (Ancestors)

AcidosisAcid-Base ImbalanceMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

ChloroacetatesAcetatesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsHydrocarbons, ChlorinatedHydrocarbons, HalogenatedHydrocarbons

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
PREVENTION
Sponsor Type
OTHER

Study Record Dates

First Submitted

February 26, 2002

First Posted

February 27, 2002

Study Start

September 1, 2001

Study Completion

September 1, 2005

Last Updated

March 25, 2015

Record last verified: 2002-01

Locations

US(1)