Santhera Targets New Indication with its Lead Compound
SNT-MC17 (idebenone) enters Phase IIa study in Duchenne muscular dystrophy
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Santhera Pharmaceuticals AG announced that it has started a Phase IIa clinical study with its lead product SNT-MC17 (idebenone) to evaluate its benefits in the treatment of Duchenne muscular dystrophy (DMD). DMD is the most common and devastating type of muscular dystrophy, causing weakness and muscle wasting in young boys for which there is no cure or effective treatment. This is a new potential indication for Santhera's SNT-MC17 (idebenone), which is expected to enter Phase III clinical trials in Europe later this year for Friedreich's ataxia, another life-threatening neuromuscular disease.
The Phase IIa DMD study is a double-blind, randomised, placebo-controlled trial which aims to assess the efficacy of SNT-MC17 in 10 to 16 year old males with cardiac dysfunction associated with DMD. The primary endpoint is to evaluate cardiac function improvement in DMD patients after one year of treatment. The effect of SNT-MC17 on muscle strength in the limbs and respiratory muscles will also be assessed as secondary endpoints. This study will take place at the University of Leuven in Belgium, and will enroll a total of 21 patients. The principal investigator of this study is Prof. Gunnar Buyse, a distinguished physician in the field of neuromuscular disease.
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