Phase III trial of L-glutamine meets endpoints for Sickle Cell Disease - Emmaus

Preliminary top-line results of a Phase III clinical trial evaluating the safety and efficacy of L-glutamine treatment, from Emmaus, for Sickle Cell Anaemia and Sickle Beta-0 Thalassemia met both the primary and secondary endpoints of the clinical trial. The prospective, randomized, double-blind, placebo-controlled, parallel-group, multi-center clinical trial enrolled 230 adult and pediatric patients as young as five years of age, across 31 US sites.

For the primary endpoint, top-line data based on an initial analysis revealed a statistically significant 25 percent reduction in the median frequency of sickle cell crises over a 48-week time period. For the secondary endpoint, top-line data based on an initial analysis also showed a statistically significant 33 percent reduction in the median frequency of hospitalizations over a 48-week time period. Both adult and pediatric patients receiving treatment demonstrated improvement. Furthermore, the therapy demonstrated a well-tolerated safety profile. The company intends to submit a New Drug Application to the FDA in mid-2014.