Theme 09 - Clinical Trials and Trial Design
Abstract
Background: The safety and efficacy of sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodoxicol- taurine) coformulation in ALS were evaluated in a phase 2 US multicenter trial (CENTAUR) encompassing both randomized (NCT03127514) and open-label extension (OLE; NCT03488524) phases. While overall treatment-emergent adverse event (TEAE) incidence was similar in the PB&TURSO versus placebo groups in the randomized phase and between those continuing versus switching to PB&TURSO in the OLE phase, treatment-emergent cardiac adverse events (TECAEs) and centrally read electrocardiographic abnormalities were reported more frequently among those receiving PB&TURSO in both phases. Objective: Describe the findings of independent expert reviews of reported TECAEs and electrocardiographic abnor- malities and of recorded electrocardiographic parameters in CENTAUR. Methods: Occurrence of TEAEs was assessed at each visit during both phases. Standard 12-lead electrocardiograms were performed at baseline in both phases and at a total of 2 visits in the randomized phase and up to 10 visits in the OLE phase, blinded and centrally read. Unblinded independent expert reviews assessed reported TECAEs and electrocar- diographic abnormalities for diagnostic accuracy and treatment emergence based on absence at baseline or lack Background: CD40L/CD40 costimulatory pathway mediates both humoral and adaptive immune responses in the pathophysiology of autoimmunity and transplant…
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