Neurological effects of encapsulated dexamethasone sodium phosphate in children aged 6-9 years with ataxia telangiectasia (NEAT): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Sam Russo, Stefan Zielen, Barbara Pietrucha et al.
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In 30 seconds
The NEAT trial was a multicentre, randomised, double-blind, placebo-controlled phase 3 study assessing the efficacy and safety of encapsulated dexamethasone sodium phosphate (eDSP) in children aged 6-9 years with ataxia telangiectasia. The study found no significant difference in the change in RmICARS score from baseline to 6 months between the eDSP and placebo groups, with a least squares mean difference of -1.30 (95% CI -2.77 to 0.18; p=0.085).
Key findings
- No significant difference in RmICARS score change between eDSP and placebo groups (least squares mean difference -1.30).
- Adverse events occurred in 92% of eDSP participants and 93% of placebo participants, with no serious treatment-related events.
- 95% of participants aged 6-9 years completed the study in both eDSP and placebo groups.
Why it matters
This study highlights the challenges in finding effective treatments for ataxia telangiectasia, a rare disorder with significant neurological implications. Understanding the efficacy and safety of potential therapies is crucial for managing this condition.