A Multi-Center, Randomized, Single-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral TTI-0102 for Treatment of Patients with Mitochondrial encephalomyopathy, lactic acidosis and strokelike episodes (MELAS).

lactic acidosis and stroke-like episodes (MELAS), Mitochondrial encephalomyopathy

The primary outcome measure will be “change in functional capacity” based upon the 12-minute walking test (12-MWT) from Day 1/Baseline to Week 24/Study Exit, Safety and tolerability endpoints include the incidence, severity and relationship of adverse events; change in clinical laboratory parameters, physical exam findings, vital signs, 12-lead electrocardiography and seizure activity.

Efficacy: Change over time as measured at Day 1/Baseline, Weeks 4, 8, 12, 16, 20 and Week 24/Study Exit: - Fatigue Severity Scale (FSS); - Quality of Life (WHOQOL-BREF), PK parameters will be determined for cysteamine, pantothenic acid (vitamin B5) and taurine, PD: Change over time as measured at Day 1/Baseline, Weeks 4, 8, 12, 16, 20 and Week 24/Study Exit for pharmacodynamic biomarkers: glutathione, glutathione disulfide, lactate, pyruvate, GDF-15, FGF21, amino acid panel (including alanine).

No related papers found yet.

France, Netherlands