Stealth BioTherapeutics announced the initiation of TAZPOWER, a Phase 2/3 study evaluating elamipretide in patients with Barth syndrome. Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene, and characterized by cardiac abnormalities, skeletal muscle weakness, recurrent infections and delayed growth.
"The severe problems experienced by patients with Barth syndrome are caused by misshapen and dysfunctional mitochondria, which reduce the energy production in the affected tissues. The resulting muscle weakness can lead to severe fatigue, heart failure and death," said Stealth Chief Medical Officer Doug Weaver. "In this study, we hope to show that elamipretide may have clinical benefit by improving function in these affected mitochondria."
TAZPOWER is a randomized, double-blind, placebo-controlled crossover study that will evaluate the effects of daily elamipretide treatment in a minimum of 12 patients with genetically confirmed Barth syndrome. Patients will be randomized to one of two sequence groups: 12 weeks of single daily subcutaneous injections of elamipretide in Treatment Period 1, followed by 12 weeks of treatment with placebo in Treatment Period 2, with a four-week wash-out period between periods, or vice versa. The primary endpoint is change in distance walked during the six-minute walk test. Secondary endpoints include functional assessments, patient-reported outcomes and safety.
"Our understanding of Barth syndrome and how it manifests has evolved significantly, but current treatment efforts are still limited to the management of symptoms," said Hilary Vernon, M.D., Ph.D., assistant professor of Pediatrics at McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins University and the primary investigator for the study. "The initiation of TAZPOWER represents an important milestone in the potential development of a disease-specific treatment option."