Isis recently announced the start of CHERISH, a multi-center Phase III clinical study evaluating the efficacy and safety of the investigational compound, ISIS-SMNRx, in non-ambulatory children with Spinal Muscular Atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled study in approximately 117 children with SMA at study centers in the US, Canada, Europe and Asia Pacific. This study was initiated in November.
The study will enroll children who are 2-12 years of age and who have been diagnosed with Spinal Muscular Atrophy, and who pass additional screening evaluations conducted at the study site to confirm eligibility. The main inclusion/exclusion criteria for this trial are onset of signs and symptoms of SMA >6 months of age; 2-12 years of age; can sit independently but has never had the ability to walk independently; without severe scoliosis.
This is the second Phase III study for ISIS-SMNRx. In September, Isis announced the launch of ENDEAR, a Phase III clinical trial to study ISIS-SMNRx in infants with SMA type I.