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news.Raptor Pharmaceutical has concluded patient recruitment in a randomized, crossover Phase 3 clinical trial evaluating delayed-release oral formulation of cysteamine bitartrate (DR Cysteamine) in patients with nephropathic cystinosis (cystinosis).
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The pivotal Phase 3 clinical trial is designed as an outpatient study of the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of DR Cysteamine dosed every 12 hours in patients with cystinosis, compared to the current standard of care, immediate-release cysteamine bitartrate, which requires dosing every six hours.
Raptor expects over 30 patients to complete the eight-week study protocol.
The primary endpoint of the multi-center, global clinical trial is the steady-state white blood cell (WBC) cystine levels of patients taking DR Cysteamine compared to immediate-release cysteamine bitartrate.
However, the secondary endpoints are the safety and tolerability of DR Cysteamine and the comparability of steady-state PK of DR Cysteamine and immediate-release cysteamine bitartrate.
Raptor Pharma chief medical officer Patrice Rioux said with enrollment completed, they anticipate that they will be able to meet their goal of reporting top line data from this clinical trial in the first quarter of 2011.