At a glance
ClinicalIndex Comparison Record- ✓Confirmed clinical diagnosis of A-T with documented neurological signs (incoordination in lateral gaze, gait ataxia with narrow base).
- ✓Autonomous gait or assisted by periodic use of support; ICARS Walking Capacities score Item 1 between 0-4.
- ✓Proven genetic diagnosis of A-T confirmed by prior documentation or central laboratory testing.
- ✓Age at least 6 years and body weight >15 kg.
- ✕CD4+ lymphocyte count <400/mm³ (age 6 years) or <150/mm³ (age >6 years); if oral candidiasis present, threshold is <200/mm³.
- ✕Blood loss ≥250 mL within 4 weeks prior to screening.
- ✕Active neoplastic disease or prior malignancy not in remission for at least 2 years.
- ✕History of severe immunological impairment.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients With Ataxia Telangiectasia
In Brief
A Phase 3 clinical trial evaluating EryDex Low dose DSP, EryDex High dose DSP, and 1 other intervention for Nervous System Disease and Genetic Syndrome. Completed, enrolled 176 participants across 22 sites in 12 countries.
Detailed Summary
Objectives: The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on Neurological Symptoms in Patients With Ataxia Telangiectasia. Initial Double-Blind Treatment Period (0 to 6 Months) Primary Efficacy Objective: • Evaluate the effect of EryDex at two dose levels (low dose and high dose DSP/infusion), compared to placebo, on central nervous system (CNS) symptoms measured by the change in the Modified International Cooperative Ataxia Rating Scale (mICARS) from baseline to Month 6 (Visit 9) in patients with ataxia telangiectasia (A-T). Secondary Efficacy Objectives: * Evaluate the effect of EryDex, compared to placebo, on the Clinical Global Impression of Change (CGI-C) in patients with A-T from baseline to Month 6 (Visit 9). * Evaluate the effect of EryDex, compared to placebo, on measures of Clinical Global Impression of Severity (CGI-S; structured) in patients with A-T from baseline to Month 6 (Visit 9) * Evaluate the effect of EryDex, compared to placebo, on measures of Adaptive behavior measures in patients with A-T by the Vineland Adaptive Behavior Scales (VABS) from baseline to Month 6 (Visit 9). Safety Objectives: • Evaluate the safety and tolerability of two non-overlapping doses of EryDex, compared to placebo, in patients with A-T over the 12-month double-blind study duration. Extension Treatment Period (6-12 Months): Primary Objective: • Evaluate the efficacy of EryDex at two dose levels (low dose and high dose DSP/infusion) compared to placebo, in treating CNS symptoms in A-T patients during longer-term treatment (up to 12 months), as measured by the mICARS. Secondary Objectives: * Evaluate the longer-term (up to 12 months) safety and tolerability of EryDex in A-T patients. * Compare the effects of EryDex on the CGI-C and CGI-S (structured), VABS, and QoL using the EQ-5D-5L scale.
Study Details
Timeline
Interventions
EDS-EP dose range of \~5-10 mg DSP/infusion
EDS-EP dose range of \~14-22 mg DSP/infusion
EDS processed autologous erythrocytes using a sodium chloride \[NaCl\] solution.