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Could red blood cells deliver a steroid to ease a rare brain disorder?

NCT ID NCT06193200

Completed Disease control Sponsor: Quince Therapeutics S.p.A. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 3 trial tested a treatment called EryDex for people with ataxia telangiectasia (A-T), a rare genetic disease that affects movement and coordination. EryDex is a steroid (dexamethasone) packaged inside the patient's own red blood cells and given as an IV infusion every 28 days. The study enrolled 105 participants to see if it could improve neurological symptoms compared to a placebo.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

EryDex (dexamethasone sodium phosphate encapsulated in the patient's own red blood cells)

What this could lead to

If it works, this could provide a way to deliver steroids directly to the body with fewer side effects, potentially easing movement and coordination problems in ataxia telangiectasia.

What could go wrong

This is a Phase 3 trial, but results are not yet known. The treatment involves regular IV infusions and may not work for everyone. Steroids can have side effects, even when encapsulated.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

ataxia telangiectasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ATAXIA TELANGIECTASIA

Contacts and locations

Locations

  • Cincinnati Children's Hospital, Division of neurology

    Cincinnati, Ohio, 45229, United States

  • Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology

    Copenhagen, 2100, Denmark

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

    London, WC1N 1DZ, United Kingdom

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

    Barcelona, 08035, Spain

  • Hospital Universitario La Paz, Department of pediatric neurology

    Madrid, 28046, Spain

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

    Frankfurt, 60596, Germany

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

    Warsaw, 04-736, Poland

  • MedPolonia sp zoo

    Poznan, 60-693, Poland

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

    Nottingham, NG7 2UH, United Kingdom

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

    Oslo, 0372, Norway

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

    Roma, 00161, Italy

  • Spedali Civili di Brescia, Pediatric immunology department

    Brescia, 25123, Italy

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

    London, SW17 0RE, United Kingdom

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

    Baltimore, Maryland, 21289, United States

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

    Houston, Texas, 77030, United States

  • University Children's Hospital Zürich - Eleonore Foundation

    Zurich, CH-8008, Switzerland

  • University Hospital Frankfurt, Pediatric and Adolescent Clinic

    Frankfurt, 60590, Germany

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B152GW, United Kingdom

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

    Los Angeles, California, 90095, United States

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