Barth syndrome is a rare genetic condition characterized by muscle weakness, cardiac abnormalities often leading to heart failure, recurrent infections, delayed growth, and reduced life expectancy.
In March 2018, Stealth completed enrollment in the TAZPOWER clinical trial, a phase 2/3 crossover study evaluating the effects of daily treatment with elamipretide in 12 patients with genetically confirmed Barth syndrome followed by an open-label treatment extension.
Primary endpoints include change in distance walked during the six-minute walk test and change in total fatigue as measured by a patient-reported outcome measure, the Barth Syndrome Symptom Assessment. Secondary endpoints include additional functional assessments, patient-reported outcomes and safety.
Privately held Stealth is focused on the development of therapeutics for diseases involving mitochondrial dysfunction.
Based on encouraging preclinical and early clinical data, the company is investigating its lead product candidate, elamipretide, in three primary mitochondrial diseases: PMM, Barth syndrome, and Leber's hereditary optic neuropathy, as well as in heart failure and dry age-related macular degeneration.
TiumBio files Clinical Trial Application to start Phase 1b TU7710 study
Moderna reports positive interim results from next-generation COVID-19 vaccine trial
Scancell initiates SCOPE trial's iSCIB1+ cohort
Valneva launches Phase 1 trial for next-generation Zika vaccine
argenx obtains VYVGART approval for primary immune thrombocytopenia in Japan
Bio-Thera Solutions' BAT8006 phase II Study receives US FDA IND approval