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

Last updated: June 6, 2025
Sponsor: Quince Therapeutics S.p.A.
Overall Status: Active - Recruiting

Phase

3

Condition

Friedreich's Ataxia

Dyskinesias

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: 106
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 3
Study Start date:
June 24, 2024
Estimated Completion Date:
September 30, 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

    Active - Recruiting

  • 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

    Active - Recruiting

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

    Frankfurt, 60596
    Germany

    Active - Recruiting

  • University Hospital Frankfurt, Pediatric and Adolescent Clinic

    Frankfurt, 60590
    Germany

    Active - Recruiting

  • Spedali Civili di Brescia, Pediatric immunology department

    Brescia, 25123
    Italy

    Active - Recruiting

  • 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

    Active - Recruiting

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

    Oslo, 0372
    Norway

    Active - Recruiting

  • MedPolonia sp zoo

    Poznań, 60-693
    Poland

    Active - Recruiting

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

    Warsaw, 04-736
    Poland

    Active - Recruiting

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

    Barcelona, 08035
    Spain

    Active - Recruiting

  • Hospital Universitario La Paz, Department of pediatric neurology

    Madrid, 28046
    Spain

    Active - Recruiting

  • University Children's Hospital Zürich - Eleonore Foundation

    Zürich, CH-8008
    Switzerland

    Active - Recruiting

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B152GW
    United Kingdom

    Active - Recruiting

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

    London, WC1N 1DZ
    United Kingdom

    Active - Recruiting

  • 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

    Active - Recruiting

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

    Nottingham, NG7 2UH
    United Kingdom

    Active - Recruiting

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

    Los Angeles, California 90095
    United States

    Active - Recruiting

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

    Baltimore, Maryland 21289
    United States

    Active - Recruiting

  • 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

    Active - Recruiting

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

    Houston, Texas 77030
    United States

    Active - Recruiting

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