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

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Primary Purpose

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

sodium dichloroacetate

About this trial

This is an interventional treatment trial for Lactic Acidosis focused on measuring inborn errors of metabolism, lactic acidosis, rare disease

Eligibility Criteria

3 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

University of California San Diego School of Medicine

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT00004493

First Posted

October 18, 1999

Last Updated

March 24, 2015

Sponsor

University of California, San Diego

1. Study Identification

Unique Protocol Identification Number

NCT00004493

Brief Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2000

Overall Recruitment Status

Completed

Study Start Date

September 1998 (undefined)

Primary Completion Date

undefined (undefined)

Study Completion Date

September 2003 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor

University of California, San Diego

4. Oversight

5. 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.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Lactic Acidosis

Keywords

inborn errors of metabolism, lactic acidosis, rare disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Enrollment

75 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

sodium dichloroacetate

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Months

Accepts Healthy Volunteers

No

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Bruce Barshop

Organizational Affiliation

University of California, San Diego

Official's Role

Study Chair

Facility Information:

Facility Name

University of California San Diego School of Medicine

City

La Jolla

State/Province

California

ZIP/Postal Code

92093-0652

Country

United States

Lactic Acidosis

About this trial

Eligibility Criteria

Sites / Locations

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

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