Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients

This was an international, multi-center, one-year, randomized, prospective, double-blind, placebo-controlled, phase III study. This study was divided into three periods: Screening (Days -30 to -1), 6-month Initial Treatment Period (Months 1-6; Visits 1-9), and 6-month Extension Treatment Period (Months 7-12; Visits 10-15). A total of 175 patients, of the 180 planned, met all selection criteria at baseline, and were randomized in a 1:1:1 fashion to one of the two EDS-EP dose levels or placebo. These patients were randomly assigned to receive one of the two doses of EDS-EP or placebo, as follows: * Group 1: EDS-EP dose range of \~5-10 mg DSP/infusion (low dose), 59 pts * Group 2: EDS-EP dose range of \~14-22 mg DSP/infusion (high dose), 57 pts * Group 3: Placebo EDS infusion, 59 pts The initial 6-month treatment period was considered complete when the endpoint assessment (at Visit 9/Month 6 or at early discontinuation) was performed for all patients. All patients who completed the assessments over the initial 6 months of the trial were eligible to continue in an additional 6-month, double-blind, placebo- controlled extension, designed to collect information on the longer-term safety and efficacy of the trial treatments. Following completion of the 6-month Initial Treatment Period, patients that met all entry criteria were re-randomized and treated as follows: * Patients originally randomized to one of the two dose levels of EryDex (low dose or high dose; Groups 1 or 2) continued in the same treatment arm. * Patients originally randomized to the Placebo group (Group 3) were re-allocated as defined at the initial randomization in equal proportions (1:1) and received either the EryDex low dose or high dose as follows: * Following 6 months of treatment, one third of the placebo patients were switched to treatment with EryDex, as described above. * After 9 months of treatment, another third of the placebo patients were switched to treatment with EryDex, as described above. * At 12 months, all remaining placebo patients were eligible to switch to open-label treatment with EryDex, as described above.