Santhera Starts European Phase III Study with its Lead Compound SNT-MC17 (idebenone) in Friedreich's Ataxia

16-Dec-2005

Santhera Pharmaceuticals AG announced that it has started a European Phase III clinical study with its lead product SNT-MC17 (idebenone) in Friedreich's ataxia (FRDA). The design of this study reflects the company's discussions with EMEA which took place earlier this year and will evaluate both the cardiac and neurological benefits of SNT-MC17 (idebenone).

The Phase III trial with SNT-MC17 (idebenone) is a double-blind, randomised, placebo-controlled study to determine its efficacy, safety and tolerability in the treatment of Friedreich's Ataxia patients with hypertrophic cardiomyopathy. The study will be performed in Germany, the UK and the Netherlands with possible extension to other EU territories. It will involve approximately 10 study centers and about 200 patients are expected to be enrolled. Patients will receive SNT-MC17 (idebenone) for a period of one year. Recruitment of patients into the trial has already started.

The Phase III study will compare the efficacy of three different doses of SNT-MC17 (idebenone) with placebo on the left ventricular mass index of FRDA patients. Hypertrophic cardiomyopathy, as measured by increased left ventricular mass indices is a prominent factor in both the morbidity and mortality of FRDA patients. In parallel this study will assess the efficacy of SNT-MC17 (idebenone) on the neurological symptoms of FRDA. This will be determined by measuring the changes in the International Cooperative Ataxia Rating Scale (ICARS).

In addition to the Phase III study, Santhera has an on-going collaboration with the National Institute of Health in the USA for FRDA and plans to initiate its own Phase III studies in the US next year for this indication with SNT-MC17 (idebenone). Santhera is also currently running a Phase IIa study in Belgium investigating the efficacy of SNT-MC17 (idebenone) for an additional neuromuscular disease indication, Duchenne Muscular Dystrophy.

https://www.bionity.com/en/news/50933/santhera-starts-european-phase-iii-study-with-its-lead-compound-snt-mc17-idebenone-in-friedreich-s-ataxia.html