Neurological Effects of EryDex on subjects with Ataxia Telangiectasia (NEAT)
Research type
Research Study
Full title
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia.
IRAS ID
1009239
Contact name
Irene Maccabruni
Contact email
Sponsor organisation
EryDel S.p.A.
Clinicaltrials.gov Identifier
Research summary
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).REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
24/YH/0004
Date of REC Opinion
12 Feb 2024
REC opinion
Further Information Favourable Opinion