Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access (CLA)

University of Florida

Status

Conditions: Mitochondrial Enzyme Deficiencies

Treatments: Drug: Dichloroacetate

Study type: Expanded Access

Funder types: Other

Identifiers

NCT01797276

IRB#420-2005

Details and patient eligibility

About

Expanded access to DCA as continued treatment for congenital lactic acidosis.

Full description

Subjects who participated in a Phase 3 Randomized Controlled Trial of DCA for treatment of Congenital Lactic Acidosis (CLA) are eligible to continue treatment with investigational medication DCA at the same dose of 25mg/kg/day for expanded access. Study participants must travel to the study site for bi-annual evaluation by the study investigator. Bi-annual evaluation will include an interim medical history review, physical exam, blood collection for DCA trough level, and urine pregnancy testing (if indicated).

Sex: Female

Ages: 18 to 50 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

• Diagnosis of congenital lactic acidosis
• Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria:
• Intolerance to DCA