Kaufmann 2006.
| Methods | Randomised, placebo controlled cross‐over study | |
| Participants | 30 participants with m.3243A>G | |
| Interventions | 25mg/kg/day of dichloracetate for 12 months | |
| Outcomes | GATE, lactate measurements in venous blood, CSF and measured by 1H‐MRS. Nerve conduction tests were used to monitor safety | |
| Notes | No benefit The trial was discontinued due to peripheral nerve toxicity |
|
| Risk of bias | ||
| Bias | Authors' judgement | Support for judgement |
| Random sequence generation (selection bias) | Low risk | "Based on a computer‐generated randomization list" |
| Allocation concealment (selection bias) | Low risk | "the research pharmacy at the University of Florida mailed study medication for each participant to the clinical investigators" |
| Incomplete outcome data (attrition bias) All outcomes | Low risk | Incomplete outcome data for some participants, although this was due to withdrawal of participants due to side‐effects in the DCA group, and early termination of the study |
| Selective reporting (reporting bias) | Low risk | All outcome measures were reported for all participants up to the termination of the trial |
| Other bias | Low risk | None identified |
| Blinding of participants and personnel (performance bias) All outcomes | Low risk | "clinical researchers remained blinded to treatment assignment throughout the trial" |
| Blinding of outcome assessment (detection bias) All outcomes | Low risk | Participants were randomly assigned to start in either the DCA or placebo arm |