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

This study has been completed.
Sponsor:
Collaborator:
University of California, San Diego
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004493
First received: October 18, 1999
Last updated: July 9, 2008
Last verified: May 2000
  Purpose

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 Intervention Phase
Lactic Acidosis
Drug: sodium dichloroacetate
Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

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

Estimated Enrollment: 75
Study Start Date: September 1998
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.

  Eligibility

Ages Eligible for Study:   3 Months and older
Genders Eligible for Study:   Both
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
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: 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

No publications provided

ClinicalTrials.gov Identifier: NCT00004493     History of Changes
Other Study ID Numbers: 199/14274, UCSD-FDR001481
Study First Received: October 18, 1999
Last Updated: July 9, 2008
Health Authority: United States: Federal Government

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

ClinicalTrials.gov processed this record on July 29, 2014