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New treatment shows promise for rare neurological disorder

NCT ID NCT06193200

First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 27 times

Summary

This Phase 3 trial tested a treatment called EryDex for people with Ataxia Telangiectasia (A-T), a rare disease that affects movement and the immune system. The study involved 105 participants who received either EryDex or a placebo every 28 days. The goal was to see if EryDex could help control neurological symptoms like coordination problems.

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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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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

Conditions

Explore the condition pages connected to this study.