Psyadon Pharmaceuticals has enrolled the first patients in a multicentre, double-blind, randomised three-way crossover Phase III study of ecopipam for the treatment of self-injurious behaviors in patients with Lesch-Nyhan disease, a rare inherited disorder.
Ecopipam, a first-in-class molecule, selectively blocks the actions of the neurotransmitter dopamine at D1 receptors, in contrast to other dopamine antagonists and agonists which act at D2 receptors.
Psyadon Pharmaceuticals president and CEO Dr Richard Chipkin said; "The self-injurious behaviors seen in these patients are devastating and in some cases sufficiently severe to require full-time restraint in specially designed wheelchairs."
The study, PSY102, will enrol patients from all over the world to assess the efficacy and safety of ecopipam in patients with Lesch-Nyhan disease with moderate-to-severe self-injurious behaviours.
The study will treat patients with either ecopipam (50mg or 100 mg/day depending on body weight) or placebo before bedtime over an 18-week period.
Behaviour Problems Inventory (BPI), a standard rating scale, will be used to assess self-injurious behaviour frequency and severity, according to the company.
The company said safety and tolerability of the doses used in the Phase III study were first tested in an open-label study, which evaluated whether patients with Lesch-Nyhan disease had a peculiar sensitivity to the drug, and established that the drug is well-tolerated and has a similar side effect profile as seen in other patient populations.
Study principal investigator Dr Jinnah said the international study is the largest therapeutic intervention ever attempted in patients with Lesch-Nyhan disease.
"If positive, it will provide relief for hundreds of sufferers who have almost no good options," Jinnah said.