Long-term safety of dichloroacetate in congenital lactic acidosis
9 evidence topics · 1 source
Objective
Location and study date
No evidence found.
Study design
Eligibility criteria
Prior study participation
Treatment
Study outcomes
Rationale for using blood lactate as a biochemical outcome
No evidence found.
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
No evidence found.
Results
Participant disposition
No evidence found.
Baseline characteristics
Pyruvate dehydrogenase complex deficiency subgroup
Efficacy results
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Dose reduction or temporary discontinuation following nerve-conduction changes
Study limitations
Summary
This open-label safety cohort comprised 8 participants, including 3 with PDC deficiency, who had participated in earlier dichloroacetate trials. Treatment continued for 9.7 to 16.5 years. The selected long-term cohort and lack of a concurrent randomized comparison limit causal conclusions about efficacy or comparative safety. Stable laboratory measures did not exclude peripheral nerve findings that prompted dose reduction or temporary discontinuation in 3 participants.