Date: 2012-02-29
Type of information:
phase: 2
Announcement: results
Company: EryDel (Italy)
Product: EryDex® (solution of dexamethasone sodium phosphate for encapsulation into human erythrocytes.)
Action mechanism:
Disease: ataxia telangiectasia
Therapeutic area: Rare diseases
Country: Italy
Trial
details: The 6-month prospective trial enrolled 22 patients with genetically confirmed AT, in two University based sites in Italy. Patients received at monthly intervals EryDex (dexamethasone sodium phosphate) therapy delivered by loading the drug into their red blood cells (RBCs) using EryDel’s patented technology (autologous approach).
The Primary efficacy measure was the International Cooperative Ataxia Rating Scale (ICARS: a rating scale completed by a trained neurologist), while the Secondary measures included the Vineland Adaptive Behavioural Scale (VABS: completed based on caregiver input). The safety of the treatment was assessed by monitoring laboratory examination, ECG, physical examination, vital signs and adverse events.
Latest
news: EryDel, a Biotech Company specialized in the delivery of innovative therapies through autologous erythrocytes, using a proprietary device technology, has announced results of a recently completed Phase II Proof of Concept study in patients with Ataxia Telangiectasia (AT), a rare progressive neurological autosomal recessive disorder that leads to mortality in most patients at an early age.
Results for the Primary efficacy measure (change in total ICARS score) in the Intent to Treat (ITT) population indicated a statistically significant reduction in the total score (p=0.02). Similarly, total score of the VABS indicated a significant improvement associated with EryDex treatment in the ITT population (p<0.0001).
Overall, treatment with EryDex was well tolerated by the patients. Only 4 patients terminated treatment prematurely (consent withdrawal: 1, protocol violation: 1, laboratory changes: 2). The most frequent adverse events included infections, fever, cough etc., and were representative of the illnesses/symptoms experienced by the patients prior to entry into the study, and generally resolved during treatment with EryDex. None of these adverse events were considered related to the study medication.
EryDel plans to discuss these results with regulatory authorities, and develop plans for additional trials that would lead to filing for regulatory approval worldwide.
