The study (IEDAT-02-2015) has been designed as a multi-center, one-year, randomized, prospective, double blind, placebo-controlled, phase III trial, to assess the effect of two non-overlapping dose ranges of EryDex, administered by IV infusion once per month, on neurological symptoms of patients with AT. Following completion of 6 months in the trial, all patients who completed the assessments as designed continued in an additional 6-month, double-blind, placebo-controlled extension, to collect information on the long-term safety and efficacy of the trial treatments.
The study involved 22 study sites in 12 different countries: 90 local operators have been trained, about 10000 EryKit_01 for patient treatment have been assessed, 175 patients have been enrolled, 1773 treatments have been delivered. Despite the COVID emergency, 132 patients reached six months of treatment (efficacy evaluation step) and 108 twelve months of treatment (safety evaluation step).
The data analysis conducted after the six-month efficacy evaluation period showed significant efficacy of the treatment on the group of patients 6 to 9 years old when compared with placebo. No clinically meaningful differences in Severe Adverse Events (SAEs) were observed between treated groups and placebo. A request to FDA for a Type B Pre-NDA Meeting has been submitted.
Along the clinical study, a total of 474 blood samples were handled and MiniATM was quantified by PCR. The target was detected in almost 50% of tested specimens. Statistical analysis revealed a collation between some ICARS scores and the amount of miniATM at screening visit. Dexamethasone effects on miniATM and patients’ outcomes are still under investigation.
A-T Nest scale was validated based on taped neurologic exams from 477 patients, ranging in age from 5 to 62 years. Based on data collected in the clinical study A-T nest have shown a convincing statistical correlation with full ICARS and derived scales; a multivariate analysis shows A-T NEST cluster Learning and Power domains isolate from the overall data variability, improving the description of a patient’s neurologic status.
