Oral dichloroacetate in chronic congenital lactic acidosis
5 evidence topics · 1 source
Objective
Location and study date
No evidence found.
Study design
Eligibility criteria
No evidence found.
Treatment
Dose per administration
Study outcomes
Rationale for using blood lactate as a biochemical outcome
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
Not applicable.
Results
Participant disposition
No evidence found.
Baseline characteristics
Efficacy results
Blood lactate response in the first participant
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Drug accumulation in the first participant
Study limitations
Summary
The report describes two participants of different ages, without a randomized comparator. Reduced blood lactate in the first participant is a biochemical observation, not evidence of a general clinical treatment effect. Reported drug accumulation limits interpretation of biochemical response without considering exposure and toxicity.