US-based Stealth BioTherapeutics has started a Phase II/III TAZPOWER clinical trial to investigate its product candidate elamipretide to treat patients with Barth syndrome caused due to TAZ gene mutations.
Designed for subcutaneous, intravenous or topical ophthalmic delivery, Elamipretide targets the inner mitochondrial membrane to restore electron transport, improve production of cellular energy and minimise oxidative stress.
The randomised, double-blind, placebo-controlled cross-over trial will assess daily elamipretide in approximately 12 patients whose disease is genetically confirmed.
Patients will be administered with subcutaneous elamipretide for 12 weeks and placebo for additional 12 weeks with a four week wash-out period in between.
The trial’s primary endpoint is change in distance walked during the six-minute walk test, while the secondary endpoints are safety, functional assessments and patient-reported outcomes.
Stealth BioTherapeutics CEO Reenie McCarthy said: "This study underscores our commitment to develop elamipretide for the treatment of rare genetic mitochondrial diseases.
"The cardiovascular and skeletal muscle symptoms affecting this population share a common thread with symptoms experienced in diseases commonly associated with aging, such as heart failure, in which mitochondrial dysfunction contributes to the clinical pathology."
Eamipretide is being further studied in two other trials for primary mitochondrial diseases Leber's hereditary optic neuropathy (LHON) and primary mitochondrial myopathy (PMM), for which it received fast-track designation from the US Food and Drug Administration (FDA) in December 2015.
The product candidate is also undergoing evaluation for the treatment of heart failure, Fuchs' corneal dystrophy and dry age-related macular degeneration.