Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access

NCT ID: NCT01797276

Last Updated: 2017-08-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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

Detailed Description

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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).

Conditions

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Mitochondrial Enzyme Deficiencies

Interventions

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Dichloroacetate

Subjects are given DCA 25mg/kg in a divided dose twice a day.

Intervention Type DRUG

Other Intervention Names

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DCA

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of congenital lactic acidosis

Exclusion Criteria

* Intolerance to DCA
Minimum Eligible Age

18 Years

Maximum Eligible Age

50 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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University of Florida

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Peter W. Stacpoole, PhD, MD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

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University of Florida Box 100226

Gainesville, Florida, United States

Site Status

Countries

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United States

Other Identifiers

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IRB#420-2005

Identifier Type: -

Identifier Source: org_study_id

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