Experimental drug aims to boost energy in rare genetic disorders

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participants must be aged 18 to 65 years * A confirmed molecular diagnosis of MELAS or LHON-Plus * Symptomatic participants with MELAS harboring the m.3243A\>G variant or a mitochondrial pathogenic variant solely mapping in a mitochondrial gene encoding a mitochondrial subunit of Complex I * Symptomatic participants with LHON-Plus harboring the m.11778G\>A or a mitochondrial pathogenic variant only mapping in a mitochondrial gene encoding a mitochondrial subunit of Complex I * Normal enzymatic Complex II activity * Participants able to swallow capsules and comply with the requirements of the study according to the opinion of the investigator * Able to give written, informed consent * Participants who are sexually active and/or fertile must use an effective birth control during the study Exclusion Criteria: * • History of another primary mitochondrial disorder * Participants acutely ill * Positive urine pregnancy test for female subjects of childbearing potential within seven days prior to the first dose of the investigational drug * Pregnancy and/or breastfeeding * Participating in another mitochondrial disorder trial * Participated in another mitochondrial disorder trial within the last six months * On a current therapy with other investigational agents * Absence of neurological symptoms, muscle weakness, or exercise intolerance * Presence of any of the following signs or symptoms in the past six months at grade 3 or higher based on the CTCAE version 4.03: nausea, vomiting, diarrhea, hypoglycemia, hyperglycemia, dizziness, blurred vision, or syncope * A known hypersensitivity to any excipient contained in the drug formulation * Current abuse of drugs and/or alcohol * Unable to consent for themselves * Participants with an enteral feeding tube * Inability to travel to the study site