Dexamethasone Trial
Investigator:
Luciana Chessa, MD
Location:
Italy
Purpose:
This open label (non-placebo controlled) study was performed to assess the safety and tolerability of the EryDex System in patients with A-T, and examine the ability of the EryDex System to reduce the neurological symptoms associated with A-T.
EryDex System:
EryDex is dexamethasone sodium phosphate loaded patient erythrocytes (red blood cells or RBCs). RBCs are removed from the patient, mechanically loaded with dexamethasone, then re-infused back into the patient. The EryDex System allows the slow release of dexamethasone, thus maintaining a sustained, low dose of drug in the blood stream for a one month period after loading.
Conclusions
The EryDex system significantly reduced the neurological symptoms associated with A-T as measured by the International Cooperative Ataxia Rating Scale (ICARS). No steroid side effects were observed.
Reference:
Chessa, L., Leuzzi, V., Plebani, A., Soresina, A., Micheli, R., D’Agnano, D., Venturi, T., Molinaro, A., Fazzi, E., Marini, M., et al. (2014). Intra-erythrocyte infusion of dexamethasone reduces neurological symptoms in ataxia teleangiectasia patients: results of a phase 2 trial. Orphanet J Rare Dis 9, 5.