The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Cytokinetics initiates a phase 2 clinical trial of CK-2127107

    Cytokinetics has initiated enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV, in California, USA.

    The clinical trial is designed to assess the effect of CK-2127107 on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA. In collaboration with Astellas, Cytokinetics is developing CK-2127107 as a potential treatment for people living with SMA and certain other debilitating neuromuscular and non-neuromuscular diseases and conditions associated with skeletal muscle weakness and/or fatigue.

    The primary objective of this double-blind, randomised, placebo-controlled clinical trial is to determine the potential pharmacodynamic effects of a suspension formulation of CK-2127107 following multiple oral doses in patients with Type II, Type III, or Type IV SMA. Secondary objectives are to evaluate the safety, tolerability and pharmacokinetics of CK-2127107. The trial will enroll 72 patients in 2 sequential, ascending dose cohorts (two cohorts of 36 patients each, half ambulatory and half non-ambulatory). Each cohort will be stratified by ambulatory versus non-ambulatory status to receive CK-2127107 dosed twice daily for 8 weeks.


    Clinical Trials Registry

    Cytokinetics website