Liestal, Switzerland – Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on neuromuscular diseases, announced today that it has closed the recruitment for its six month pivotal Phase III trial with SNT-MC17 (INN: idebenone) in Friedreich’s Ataxia in the United States. The two study centers at the Children’s Hospital of Philadelphia and the School of Medicine of the University of California, Los Angeles, have enrolled a total of 70 patients. The strong support from patient advocacy groups was instrumental in considerably exceeding the original recruitment target of 51 individuals.
The IONIA ( Idebenone effects On Neurological ICARS Assessments) trial is a double-blind, randomized, placebo-controlled study of six months duration investigating the efficacy of two doses of SNT-MC17/idebenone compared to placebo. The primary endpoint is the change in the International Cooperative Ataxia Rating Scale (ICARS), a neurological scale, where the difference between baseline and en d of treatment for each of the dosing groups will be compared with the change in the placebo group. The IONIA study also investigates additional neurological endpoints as well as activities of daily living parameters and cardiac outcomes. The study protocol incorporates advice provided by the US Food and Drug Administration under Special Protocol Assessment. The six month treatment period will be followed by a one month follow-up period. As of today, more than twenty patients have already completed the trial and are enrolled into an open label extension study on the high dose level.