Collection of high quality muscle biopsies for use in DMD clinical trial analysis; process development and implementation


Continuous expression of utrophin protein by a utrophin modulator has potential as a disease modifying treatment for Duchenne muscular dystrophy (DMD) patients regardless of their dystrophin mutation. Summit’s lead utrophin modulator, ezutromid, is an orally administered, small molecule currently being dosed in a 48-week open label trial, PhaseOut DMD (see NCT02858362). This proof of concept study includes analyses of utrophin protein (estimate of target engagement) and muscle fibre regeneration (a potential pharmacodynamic marker) from muscle biopsy samples, one taken at baseline and another at either 24 or 48 weeks.


Leave A Reply