Controlled clinical trial of dichloroacetate for congenital lactic acidosis in children
15 evidence topics · 2 sources
Objective
Location and study date
Long-term follow-up endpoint
Study design
Eligibility criteria
No evidence found.
Treatment
Investigational dose
Study outcomes
Rationale for using global assessment of treatment efficacy as a primary endpoint
Composite clinical endpoint
Endpoint validation and component weighting
No evidence found.
Statistical analysis description
Number of participants (Planned and analyzed)
Randomized-study enrollment
Long-term follow-up cohort
Description of analysis sets
Long-term analysis approach
Results
Participant disposition
Baseline characteristics
Pyruvate dehydrogenase deficiency subgroup
Efficacy results
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Randomized-period tolerability
Study limitations
Summary
Only 11 of the 43 enrolled participants had pyruvate dehydrogenase deficiency, limiting direct applicability of the overall trial result to PDCD. The reported clinical outcomes did not improve. The subsequent open-label follow-up involved 36 participants from the earlier controlled trial and therefore does not represent an independent randomized replication. Peripheral nerve changes during long-term exposure require consideration alongside the short-term tolerability finding.