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Study on the Effects of EryDex (Dexamethasone Sodium Phosphate) for Children Aged 6-9 with Ataxia Telangiectasia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a treatment called EryDex on individuals with Ataxia Telangiectasia, a rare genetic disorder that affects the nervous system and other parts of the body. The treatment involves using a medication called Dexamethasone sodium phosphate, which is encapsulated into the patient's own red blood cells. This process is designed to help manage symptoms related to the central nervous system, such as coordination and movement difficulties.

The purpose of the study is to evaluate how effective EryDex is in improving neurological symptoms in children aged 6 to 9 years with Ataxia Telangiectasia. Participants in the study will be randomly assigned to receive either the EryDex treatment or a placebo. The study is conducted in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure the results are unbiased and reliable.

Throughout the study, participants will undergo regular assessments to monitor changes in their symptoms. The study will last for several months, with visits scheduled to track progress and gather data on the treatment's effects. The ultimate goal is to determine whether EryDex can provide meaningful improvements in the quality of life for those living with Ataxia Telangiectasia.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria, including a diagnosis of Ataxia Telangiectasia and genetic confirmation.

    Participants must be at least 6 years old and weigh at least 15 kg. Written consent is required from the participant or their legal representative.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to establish a baseline for neurological symptoms. This involves measuring the RmICARS score, which evaluates coordination and movement abilities.

  3. Step 3

    Treatment administration

    Participants receive Dexamethasone sodium phosphate encapsulated into autoerythrocytes. This is administered as a solution for infusion through an intravenous route.

    The treatment is given periodically over the course of the study, with specific intervals determined by the study protocol.

  4. Step 4

    Ongoing assessments

    Regular assessments are conducted to monitor changes in neurological symptoms. These assessments occur at scheduled visits, with the primary endpoint being evaluated at Visit 9, which is Day 168 of the study.

    The CGI-S and CGI-C scales are used to evaluate the overall clinical effect of the treatment compared to a placebo.

  5. Step 5

    Completion of the study

    The study concludes with a final assessment to determine the effect of the treatment on neurological symptoms compared to the baseline.

    The study is estimated to end by July 31, 2025, with recruitment starting on April 30, 2024.

Who can join the trial?

6 criteria

  • The patient must have a confirmed diagnosis of **Ataxia Telangiectasia (A-T)**. This means they have specific neurological signs like trouble coordinating head and eye movements or difficulty walking.
  • The patient should be able to walk on their own or with occasional help, as measured by a specific score called the **ICARS score**.
  • The patient must be at least 6 years old by the time they start the study.
  • The patient must have a genetic test that confirms they have A-T.
  • The patient must weigh at least 15 kilograms.
  • Written consent to participate in the study must be provided by the patient and their parent or caregiver, or a legal representative. If the patient is too young to give full consent, they should agree to participate as much as they can.

Who cannot join the trial?

8 criteria

  • Patients who are not diagnosed with Ataxia Telangiectasia cannot participate. Ataxia Telangiectasia is a rare genetic disorder that affects movement and coordination.
  • Patients who are not within the age range of 6 to 9 years old cannot participate.
  • Patients who are not able to follow the study procedures or attend all required visits cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are taking medications that might affect the study results cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have a history of allergic reactions to the study medication cannot participate.
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Investigated drugs

EryDex is a treatment being studied for its effects on neurological symptoms in patients with Ataxia Telangiectasia. This therapy involves using a special system to deliver a medication through red blood cells. The goal is to see if EryDex can improve symptoms related to the central nervous system in children aged 6 to 9 years old.

What is already known about the treatment

EryDex – EryDex is administered as an infusion, where the medication is delivered directly into the bloodstream. It is currently being studied in clinical trials for its potential effects on neurological symptoms associated with Ataxia Telangiectasia, a rare genetic disorder. The main therapeutic indication for EryDex is to improve central nervous system symptoms in affected individuals. At the molecular level, EryDex works by delivering dexamethasone, a corticosteroid, encapsulated in red blood cells, which may help reduce inflammation and modulate immune responses. It is classified pharmacologically as a corticosteroid therapy.

Investigated diseases

Ataxia Telangiectasia – Ataxia Telangiectasia is a rare genetic disorder that affects various systems in the body, primarily the nervous and immune systems. It is characterized by progressive difficulty with coordination and movement, known as ataxia, which often begins in early childhood. Individuals with this condition may also develop small, dilated blood vessels, called telangiectasias, particularly in the eyes and on the skin. As the disease progresses, affected individuals may experience increased susceptibility to infections due to immune system deficiencies. Neurological symptoms tend to worsen over time, leading to challenges with balance, speech, and motor skills. The condition is caused by mutations in the ATM gene, which plays a role in DNA repair and cell cycle control.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-509077-23-00Protocol codeIEDAT-04-2022Estimated enrolment114 patientsSponsorQuince Therapeutics S.p.A.

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