| Methods |
Randomised, double‐blind, placebo‐controlled parallel‐group study |
| Participants |
43 participants with congenital lactic acidosis, including 25 with one or more defects of the respiratory chain and 7 with a mutation of mtDNA |
| Interventions |
12.5 mg/kg of dichloracetate every 12 hours for 6 months |
| Outcomes |
GATE; linear growth; fasting blood lactate, and lactate after a carbohydrate meal; the frequency of severe or recurrent illnesses or hospital admissions; and safety tests of liver and peripheral nerve function |
| Notes |
Statistically significant attenuation of the rise in blood lactate following a carbohydrate meal. No clinical benefit |
| Risk of bias |
| Bias |
Authors' judgement |
Support for judgement |
| Random sequence generation (selection bias) |
Unclear risk |
Method of randomisation not specified |
| Allocation concealment (selection bias) |
Unclear risk |
Method of allocation not specified |
| Incomplete outcome data (attrition bias)
All outcomes |
Low risk |
Incomplete outcome data adequately addressed |
| Selective reporting (reporting bias) |
Low risk |
All outcome measures in methods section were reported in results section |
| Other bias |
Low risk |
None identified |
| Blinding of participants and personnel (performance bias)
All outcomes |
Low risk |
This was a double‐blind trial. "[P]lacebo solution was formulated in an identical manner [to the DCA solution], except for the absence of DCA." |
| Blinding of outcome assessment (detection bias)
All outcomes |
Low risk |
Participants were randomly assigned to placebo or DCA treatment |
