ATTeST study explores novel steroid delivery for ataxia telangiectasia

Although intra-erythrocyte delivery of dexamethasone sodium phosphate appears safe, it may not significantly improve neurological function in children with ataxia telangiectasia when compared to placebo, according to results from the ATTeST Phase 3 clinical trial.

The multicenter, double-blind trial involved 176 children aged 6 years or older with preserved gait, who were randomly assigned to receive low-dose or high-dose dexamethasone or placebo, administered intravenously once a month for 6 months.

Results showed no significant improvement in motor function, as measured by the modified International Cooperative Ataxia Rating Scale (mICARS), compared to placebo. Adverse events were common across all groups, but no severe steroid-related side effects, such as hyperglycemia or Cushingoid appearance, were observed.

Although the trial did not meet its primary efficacy endpoint, researchers suggested that treatment delays and age-related differences in response might have influenced outcomes.

Reference
Zielen S, Crawford T, Benatti L, et al. Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial. Lancet Neurol. 2024;23(9):871-882. doi: 10.1016/S1474-4422(24)00220-5. PMID: 39152028.