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

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ClinicalTrials.gov Identifier: NCT01797276
Expanded Access Status : No longer available
First Posted : February 22, 2013
Last Update Posted : August 8, 2017
Sponsor:
Information provided by (Responsible Party):
University of Florida

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

Condition or disease Intervention/treatment
Mitochondrial Enzyme Deficiencies Drug: Dichloroacetate

Detailed 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).

Study Type : Expanded Access
Expanded Access Type : Individual Patients, Treatment IND/Protocol
Official Title: Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access

Resource links provided by the National Library of Medicine



Intervention Details:
  • Drug: Dichloroacetate
    Subjects are given DCA 25mg/kg in a divided dose twice a day.
    Other Name: DCA

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

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): NCT01797276


Locations
United States, Florida
University of Florida Box 100226
Gainesville, Florida, United States, 32610-0226
Sponsors and Collaborators
University of Florida
Investigators
Principal Investigator: Peter W. Stacpoole, PhD, MD University of Florida

Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT01797276     History of Changes
Other Study ID Numbers: IRB#420-2005
First Posted: February 22, 2013    Key Record Dates
Last Update Posted: August 8, 2017
Last Verified: August 2017

Keywords provided by University of Florida:
Dichloroacetate (DCA)
Congenital Lactic Acidosis (CLA)
acidosis
orphan disease

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