Evaluate the Neurological Effects of EryDex on Subjects With A-T

Last updated: January 12, 2026
Sponsor: Quince Therapeutics S.p.A.
Overall Status: Completed

Phase

3

Condition

Dyskinesias

Friedreich's Ataxia

Spinocerebellar Disorders

Treatment

Placebo

Dexamethasone sodium phosphate

Clinical Study ID

NCT06193200
IEDAT-04-2022
  • Ages > 6
  • All Genders

Study Summary

This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangiectasia (A-T).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Clinical diagnosis of A-T

  • In autonomous gait or is helped by periodic use of a support

  • Genetic confirmation of A-T

  • Body weight ≥15 kg

Exclusion

Exclusion Criteria:

  • Participation in another clinical study

  • Immune impairment

  • History of severe impairment of the immunological system

  • Current neoplastic disease or previous neoplastic disease not in remission for atleast 2 years

  • Severe or unstable pulmonary disease

  • Uncontrolled diabetes

  • Current chronic or acute significant renal and/or hepatic impairment

  • Any previous oral or parenteral steroid use within 6 weeks before Baseline.Treatment with inhaled or intranasal steroids for asthma or allergies, as well asuse of topical steroids will be permitted

  • A disability that may prevent the subject from completing all study requirements

Study Design

Total Participants: 105
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 3
Study Start date:
June 24, 2024
Estimated Completion Date:
December 17, 2025

Study Description

The EryDex System (EDS) is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) which is infused into the patient.

In the placebo arm, the subjects will receive autologous erythrocytes prepared with the EDS process using a placebo solution.

Upon completion of all screening assessments for eligibility, subjects meeting all selection criteria at baseline will be randomized in a 1:1 fashion to EryDex or placebo. Approximately 86 subjects 6- to 9-years-old, approximately 43 per group, will be randomized. Approximately 20 subjects 10 years of age and above, 10 per treatment group, may also be enrolled.

Connect with a study center

  • UZ Leuven

    Leuven, 3000
    Belgium

    Site Not Available

  • Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology

    Copenhagen, 2100
    Denmark

    Site Not Available

  • Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology

    Copenhagen 2618425, 2100
    Denmark

    Site Not Available

  • Tübingen University Hospital, Division Translational Genomics of Neurodegenerative Diseases

    Tübingen, Bade-Würtenberg 72076
    Germany

    Site Not Available

  • Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin Kinderheilkunde III Neuropädiatrie, Allgemeinpädiatrie, Diabetologie, Endokrinologie, Sozialpädiatrie

    Tübingen, Bade-Würtenberg 72076
    Germany

    Site Not Available

  • Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin Kinderheilkunde III Neuropädiatrie, Allgemeinpädiatrie, Diabetologie, Endokrinologie, Sozialpädiatrie

    Tübingen 2820860, Bade-Würtenberg 72076
    Germany

    Site Not Available

  • IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases

    Frankfurt, 60596
    Germany

    Site Not Available

  • University Hospital Frankfurt, Pediatric and Adolescent Clinic

    Frankfurt, 60590
    Germany

    Site Not Available

  • IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases

    Frankfurt 2925536, 60596
    Germany

    Site Not Available

  • University Hospital Frankfurt, Pediatric and Adolescent Clinic

    Frankfurt 2925536, 60590
    Germany

    Site Not Available

  • Spedali Civili di Brescia, Pediatric immunology department

    Brescia, 25123
    Italy

    Site Not Available

  • Spedali Civili di Brescia, Pediatric immunology department

    Brescia 3181554, 25123
    Italy

    Site Not Available

  • Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health

    Rom, 00161
    Italy

    Site Not Available

  • Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health

    Roma, 00161
    Italy

    Site Not Available

  • Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health

    Roma 8957247, 00161
    Italy

    Site Not Available

  • Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening

    Oslo, 0372
    Norway

    Site Not Available

  • Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening

    Oslo 3143244, 0372
    Norway

    Site Not Available

  • MedPolonia sp zoo

    Poznan 3088171, 60-693
    Poland

    Site Not Available

  • MedPolonia sp zoo

    Poznań, 60-693
    Poland

    Site Not Available

  • Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic

    Warsaw, 04-736
    Poland

    Site Not Available

  • Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic

    Warsaw 756135, 04-736
    Poland

    Site Not Available

  • Hospital Universitari Vall d'Hebron, Department of pediatric neurology

    Barcelona, 08035
    Spain

    Site Not Available

  • Hospital Universitari Vall d'Hebron, Department of pediatric neurology

    Barcelona 3128760, 08035
    Spain

    Site Not Available

  • Hospital Universitario La Paz, Department of pediatric neurology

    Madrid, 28046
    Spain

    Site Not Available

  • Hospital Universitario La Paz, Department of pediatric neurology

    Madrid 3117735, 28046
    Spain

    Site Not Available

  • University Children's Hospital Zürich - Eleonore Foundation

    Zurich 2657896, CH-8008
    Switzerland

    Site Not Available

  • University Children's Hospital Zürich - Eleonore Foundation

    Zürich, CH-8008
    Switzerland

    Site Not Available

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B152GW
    United Kingdom

    Site Not Available

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham 2655603, B152GW
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children, Zayed Centre for Research

    London, WC1N 1DZ
    United Kingdom

    Site Not Available

  • Guy's and St Thomas' NHS Foundation Trust Evelina London Children's Hospital

    London, SE1 7EH
    United Kingdom

    Site Not Available

  • St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection

    London, SW17 0RE
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children, Zayed Centre for Research

    London 2643743, WC1N 1DZ
    United Kingdom

    Site Not Available

  • St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection

    London 2643743, SW17 0RE
    United Kingdom

    Site Not Available

  • Nottingham Children's Hospital, Queen's Medical Center, Children's neurology

    Nottingham, NG7 2UH
    United Kingdom

    Site Not Available

  • Nottingham Children's Hospital, Queen's Medical Center, Children's neurology

    Nottingham 2641170, NG7 2UH
    United Kingdom

    Site Not Available

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

    Los Angeles, California 90095
    United States

    Site Not Available

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

    Los Angeles 5368361, California 5332921 90095
    United States

    Site Not Available

  • The Johns Hopkins Hospital, Division of pediatric allergy and immunology

    Baltimore, Maryland 21289
    United States

    Site Not Available

  • The Johns Hopkins Hospital, Division of pediatric allergy and immunology

    Baltimore 4347778, Maryland 4361885 21289
    United States

    Site Not Available

  • Biotrial Inc.

    Newark, New Jersey 07103
    United States

    Site Not Available

  • Rare Disease Research

    Hillsborough, North Carolina 27278
    United States

    Site Not Available

  • Cincinnati Children's Hospital, Division of neurology

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Cincinnati Children's Hospital, Division of neurology

    Cincinnati 4508722, Ohio 5165418 45229
    United States

    Site Not Available

  • UT Health Houston, Department of pediatrics, division of child & adolescent neurology

    Houston, Texas 77030
    United States

    Site Not Available

  • UT Health Houston, Department of pediatrics, division of child & adolescent neurology

    Houston 4699066, Texas 4736286 77030
    United States

    Site Not Available

Map preview placeholder

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.