Table 2

Efficacy endpoints

NPretrial periodEvaluation period†P valueStatistical analysis
Annual relapse rate of focal neurological deficits102.22±0.730.72±0.620.001*t-test
Frequency of intravenous formulation with L-arginine106.94±10.541.09±2.390.1405t-test
JMDRSWilcoxon signed-rank test
 Section 1105.5 (1–11)6 (1–12)0.7969
 Section 2103.5 (0–13)4.5 (0–13)0.8125
 Section 3103 (0–4)3 (0–7)0.5
 Section 4100 (0–1)0 (0–2)1
 Section 5100.5 (0–1)1 (0–1)1
 Section 6100 (0–1)0 (0–1)1
 Section 7101.5 (0–5)2.5 (0–6)0.375
 Total scores1015 (2–28)18 (1–32)0.5625
Taurinet-test
 Plasma taurine (nmol/mL)1057.57±20.29945.67±406.180.0001*
 CSF taurine (nmol/mL)711.24±2.8842.11±13.770.0007*
Lactate and pyruvatet-test
 Serum lactate (mg/dL)1032.49±12.9735.76±12.640.4079
 CSF lactate (mg/dL)740.54±15.3145.73±17.870.4742
 Serum pyruvate (mg/dL)101.26±0.391.42±0.510.395
 CSF pyruvate (mg/dL)71.39±0.391.72±0.520.1672
 Serum lactate:pyruvate ratio1026.14±5.9225.51±4.890.7048
 CSF lactate:pyruvate ratio728.47±4.9326.03±4.680.0521
  • Data are expressed as mean±SD, except JMDRS being expressed as median (range).

  • *P<0.05.

  • †The first 8 weeks after the start of the study drug administration were not included in the evaluation period.

  • CSF, cerebrospinal fluid; JMDRS, Japanese Mitochondrial Disease Rating Scale.