Recruiting

OLE_NEATEryDex for Ataxia Telangiectasia Patients

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What is being tested

Dexamethasone sodium phosphate

Drug
Who is being recruted

Primary Immunodeficiency Diseases+19

+ Ataxia

+ Ataxia Telangiectasia

Over 6 Years
+6 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 3
Interventional
Study Start: December 2024
See protocol details

Summary

Principal SponsorQuince Therapeutics S.p.A.
Study ContactDirk Thye, MDMore contacts
Last updated: January 31, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: December 11, 2024

Actual date on which the first participant was enrolled.

This clinical study is designed to continue providing a treatment called EryDex to patients with a condition known as Ataxia Telangiectasia, who have already participated in an earlier study. Ataxia Telangiectasia is a rare genetic disorder affecting movement and immune system function. The main aim is to observe how patients respond to the treatment over a longer period. This study is important because it offers continuous treatment to those who have already been involved in previous research, potentially providing further insights into the effectiveness and safety of EryDex for managing symptoms of this challenging condition. Participants in this study will receive the EryDex treatment for 12 months. EryDex involves a special process where a medication called dexamethasone sodium phosphate is delivered inside the patient’s own red blood cells. This is done to help manage the symptoms of Ataxia Telangiectasia. Throughout the study, researchers will closely monitor the participants to ensure there are no safety issues and to gather more information on the treatment's impact on their health. By the end of the study, each participant will undergo a final safety check to complete their involvement.

Official TitleAn Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)
NCT06664853
Principal SponsorQuince Therapeutics S.p.A.
Study ContactDirk Thye, MDMore contacts
Last updated: January 31, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

105 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.



Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 6 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Primary Immunodeficiency DiseasesAtaxiaAtaxia TelangiectasiaBrain DiseasesCardiovascular DiseasesCentral Nervous System DiseasesCerebellar AtaxiaCerebellar DiseasesImmunologic Deficiency SyndromesImmune System DiseasesMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsNutritional and Metabolic DiseasesTelangiectasisVascular DiseasesNeurocutaneous SyndromesSpinocerebellar AtaxiasDyskinesiasGenetic Diseases, InbornDNA Repair-Deficiency Disorders

Criteria

2 inclusion criteria required to participate
body weight ≥15 kg

participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments

4 exclusion criteria prevent from participating
safety contraindications for continuation of treatment, as determined by the investigator

clinically significant immune impairment that, in the opinion of the Investigator, precludes further treatment with corticosteroids

Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.

requiring treatment with a systemic corticosteroid

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
intravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)

Study Objectives

Primary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 22 locations

Recruiting

MedPolonia sp zoo

Poznan, PolandOpen MedPolonia sp zoo in Google Maps
Recruiting

Great Ormond Street Hospital for Children, Zayed Centre for Research

London, United Kingdom
Suspended

University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence

Los Angeles, United States
Suspended

The Johns Hopkins Hospital, Division of pediatric allergy and immunology

Baltimore, United States
Recruiting
22 Study Centers