The study is designed to evaluate the safety and tolerability of different doses of ecopipam in adults (group 1), adolescents (group 2) and children (group 3), with LND.
Richard Chipkin, president and CEO of Psyadon Pharmaceuticals, said: "We are very excited to be starting our clinical trial with ecopipam in patients with Lesch-Nyhan Disease. There are currently no treatments available for these patients, and we are hopeful that this drug will provide some benefit."
Ecopipam is a selective antagonist of the D1-family of dopamine receptors. Psyadon licensed worldwide rights to ecopipam from Schering-Plough, an affiliate of Merck & Co, in 2008. Although an optimal clinical indication has not yet been identified, ecopipam has already been studied in over 2,000 patients.