Active Digestion, Kidneys & Other Organs NIHR-supported project Brain & Nervous System

OLE NEAT Open-Label Extension of EryDex in A-T

Funder NIHR (part of DHSC)
Recipient organisation NIHR Nottingham Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Jan 2025 — Aug 2026

In plain English

AI plain-English summary

A rare genetic disease that progressively destroys the nervous system is being tested with a treatment that packs steroids into the patient’s own red blood cells. This study addresses ataxia-telangiectasia (A-T), a condition that causes children to lose coordination, develop severe lung infections, and often die by their twenties. No approved treatment exists. The trial tests whether monthly infusions of dexamethasone—encapsulated inside red blood cells via the EryDex System—can safely deliver the drug without the toxic side effects that free steroids cause. Around 106 participants from North America, Europe, and the UK will receive 12 infusions over 14 months, with each treatment drawing 50 mL of blood, loading it with the drug, and reinfusing it. If the treatment proves safe and tolerable, it could become the first therapy to slow A-T’s progression. The approach also demonstrates a broader drug-delivery method—using red blood cells as microscopic carriers—that might be adapted for other conditions where precise, sustained drug release is needed without harming healthy tissue. For now, the immediate impact is on the children and families living with a devastating, untreatable disease.

View original technical description
The purpose of this study is to collect information on the safety and tolerability of the trial treatment. Study drug will be placed into the red blood cells using the EryDex System. About 50 mL of blood will be collected. After study drug is placed into the red blood cells using the EryDex System, the red blood cells will then be put back into the body through a vein in the arm (infusion).Approximately 106 participants from IEDAT-04-2022 will take part in this study. The study is being done in various regionsincluding North America, Europe, and United Kingdom.This study treatment period lasts approximately 14 months and will consist of 12 infusions (one infusion approximately every 28 days).This is an open-label study, which means that participants will receive the study drug. The study drug is dexamethasone sodium phosphate (DSP), encapsulated in the red blood cells, by means of a procedure calledEryDex System. The treatment is called “EryDex”. During this EryDex process, 50 mL of blood will be collected before each treatment and the subsequent infusion will be a totalvolume of approximately 70-80 mL (includes blood, study drug and solutions used to conduct the EryDex System Process).

Researchers

Andrew Prayle (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia (NEAT)
A Multi-center, Randomized, Double-blind, Placebocontrolled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia
Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
Intra Erythrocyte Dexamethasone in the treatment of Ataxia Telangiectasia
A Phase 3, Randomized, Double-Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion Dependent Anemia in Adult Participants with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS)

Plain English summaries and category classifications on this site are generated by AI and may not perfectly reflect the original research.