RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR
Status:
Completed
Trial end date:
2017-11-30
Target enrollment:
Participant gender:
Summary
Mitochondrial myopathies are a multisystemic group of disorders that are characterized by a
wide range of biochemical and genetic mitochondrial defects and variable modes of
inheritance. Currently there are no effective treatments for this disease. Despite the
heterogeneous myopathy phenotypes, a unifying feature of mitochondrial myopathies is that the
pathogenic mtDNA mutations and/or nuclear mutations of the electron transport chain
invariably lead to dysfunctional mitochondrial respiration. This reduction in mitochondrial
respiration leads to a reduced ability to produce cellular adenosine triphosphate (ATP),
often resulting in muscle weakness, exercise intolerance, and fatigue in patients with
mitochondrial myopathies.
RTA 408 is a potent activator of Nrf2 and inhibitor of NF κB (nuclear factor
kappa-light-chain-enhancer of activated B cells), and thus induces an antioxidant and
anti-inflammatory phenotype. Several lines of evidence suggest that Nrf2 activation can
increase mitochondrial respiration and biogenesis. Collectively, available data suggest that
the ability of RTA 408 to activate Nrf2 and induce its target genes could potentially improve
muscle function, oxidative phosphorylation, antioxidant capacity, and mitochondrial
biogenesis in patients with mitochondrial myopathies.
This study will be a randomized, placebo-controlled, double-blind, dose-escalation study to
evaluate the safety of omaveloxolone (RTA 408) at various doses in patients with
mitochondrial myopathies.