Back to resultsDichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access (CLA)
Primary Purpose
Mitochondrial Enzyme Deficiencies
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Dichloroacetate
Sponsored by
About this trial
This is an expanded access trial for Mitochondrial Enzyme Deficiencies focused on measuring Dichloroacetate (DCA), Congenital Lactic Acidosis (CLA), acidosis, orphan disease
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of congenital lactic acidosis
- Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria:
- Intolerance to DCA
Sites / Locations
- University of Florida Box 100226
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT01797276
First Posted
February 15, 2013
Last Updated
August 7, 2017
Sponsor
University of Florida
1. Study Identification
Unique Protocol Identification Number
NCT01797276
Brief Title
Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
Acronym
CLA
Official Title
Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
Study Type
Expanded Access
2. Study Status
Record Verification Date
August 2017
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Florida
4. Oversight
5. Study Description
Brief Summary
Expanded access to DCA as continued treatment for congenital lactic acidosis.
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mitochondrial Enzyme Deficiencies
Keywords
Dichloroacetate (DCA), Congenital Lactic Acidosis (CLA), acidosis, orphan disease
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Dichloroacetate
Other Intervention Name(s)
DCA
Intervention Description
Subjects are given DCA 25mg/kg in a divided dose twice a day.
10. Eligibility
Sex
Female
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of congenital lactic acidosis
Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria:
Intolerance to DCA
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Peter W. Stacpoole, PhD, MD
Organizational Affiliation
University of Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Florida Box 100226
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610-0226
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
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