We updated the design of this site on December 18, 2017. Learn more.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia

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.
ClinicalTrials.gov Identifier: NCT00004493
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
Sponsor:
Information provided by:

Study Description
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.


Condition or disease Intervention/treatment Phase
Lactic Acidosis Drug: sodium dichloroacetate Phase 2

Detailed Description:

PROTOCOL OUTLINE: This is a randomized, double blind, cross over study. Patients are randomized to start with one of two different doses of sodium dichloroacetate (DCA).

Patients receive one of two doses of oral DCA for 6 months, then switch to the alternate dose for 6 months. This course is repeated once.

Patients are followed for up to 2 years.

Completion date provided represents the completion date of the grant per OOPD records


Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 75 participants
Primary Purpose: Treatment
Study Start Date : September 1998
Estimated Study Completion Date : September 2003
Arms and Interventions


Outcome Measures

Eligibility Criteria

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   3 Months and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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): NCT00004493


Locations
United States, California
University of California San Diego School of Medicine
La Jolla, California, United States, 92093-0652
Sponsors and Collaborators
University of California, San Diego
Investigators
Study Chair: Bruce Barshop University of California, San Diego
More Information

ClinicalTrials.gov Identifier: NCT00004493     History of Changes
Other Study ID Numbers: 199/14274
UCSD-FDR001481
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: March 25, 2015
Last Verified: May 2000

Keywords provided by FDA Office of Orphan Products Development:
inborn errors of metabolism
lactic acidosis
rare disease

Additional relevant MeSH terms:
Acidosis
Acidosis, Lactic
Acid-Base Imbalance
Metabolic Diseases