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

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia (NEAT)

Funder NIHR (part of DHSC)
Recipient organisation NIHR Great Ormond Street Hospital Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Jan 2025 — Jul 2025

In plain English

AI plain-English summary

A monthly infusion of dexamethasone carried inside the patient’s own red blood cells is being tested against a placebo in 106 children and young adults with Ataxia Telangiectasia (A-T), a rare genetic disorder that destroys coordination and immune function. A-T progressively damages the cerebellum, leaving children unable to walk steadily, speak clearly, or fight off infections. No treatment currently slows the neurological decline. This trial tests whether EryDex—a system that loads dexamethasone sodium phosphate into red blood cells and reinfuses them—can deliver the steroid to the brain without the severe side effects of oral steroids. If EryDex works, it would be the first therapy to slow or halt the neurological deterioration of A-T. That would mean children with the condition could retain mobility, speech, and independence for years longer than they do now. The treatment would also avoid the bone thinning, growth suppression, and metabolic damage that make long-term oral steroids untenable in children. For families, the difference would be measured in months or years of functional life, not just in lab scores.

View original technical description
The purpose of this study is to see if the study treatment (EryDex) works better or is safer than taking a placebo for the neurological symptoms of Ataxia Telangiectasia. Study drug or placebo will be placed into the red blood cells using the EryDex System.About 50 mL of blood will be collected. After study drug or placebo 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 with Ataxia Telangiectasia, 6 years of age and older, will be enrolled in this study: a minimum of 86 participants 6 to 9 years-old and approximately 20 participants 10 years and older. The study is being done in various regions including North America, Europe, and United Kingdom.The participants will receive either the study drug or placebo in the same proportion (1:1) for this study. This is a double-blind study. This study treatment period lasts approximately 6 months and will consist of 6 infusions (one infusion every 28 days). The participants will receive either the study drug or placebo in the same proportion (1:1) for this study. This is a double-blind study. This study treatment period lasts approximately 6 months and will consist of 6 infusions (one infusion every 28 days). The study drug is dexamethasone sodium phosphate (DSP), encapsulated in the red blood cells, by means of a procedure called EryDex System. The treatment is called “EryDex”. The placebo is a saline solution.This study consists of the following treatment groups:· Group 1: will receive approximately 17 mg of EryDex (DSP placed into red blood cells); · Group 2: will receive placebo (placebo placed into red blood cells).During this EryDex process, 50 mL of blood will be collected before each treatment and the subsequent infusion will be a total volume of approximately 70-80 mL (includes blood, study drug/placebo and solutions used to conduct the EryDex System Process).

Researchers

Winnie Ip (Principal Investigator)

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