Breakthrough treatment trial offers hope for children with rare neurological disorder
NCT ID NCT06193200
Summary
This completed Phase 3 trial tested whether a medication called EryDex could help control neurological symptoms in children with ataxia telangiectasia, a rare genetic disorder that affects movement and coordination. The study involved 105 children aged 6 and older who received either the treatment or a placebo infusion every 28 days. Researchers measured changes in movement, balance, and overall neurological function to see if EryDex could slow disease progression.
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Contacts and locations
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Locations
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Cincinnati Children's Hospital, Division of neurology
Cincinnati, Ohio, 45229, United States
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Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
Copenhagen, 2100, Denmark
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Great Ormond Street Hospital for Children, Zayed Centre for Research
London, WC1N 1DZ, United Kingdom
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Hospital Universitari Vall d'Hebron, Department of pediatric neurology
Barcelona, 08035, Spain
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Hospital Universitario La Paz, Department of pediatric neurology
Madrid, 28046, Spain
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IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
Frankfurt, 60596, Germany
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Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic
Warsaw, 04-736, Poland
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MedPolonia sp zoo
Poznan, 60-693, Poland
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Nottingham Children's Hospital, Queen's Medical Center, Children's neurology
Nottingham, NG7 2UH, United Kingdom
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Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening
Oslo, 0372, Norway
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Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
Roma, 00161, Italy
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Spedali Civili di Brescia, Pediatric immunology department
Brescia, 25123, Italy
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St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection
London, SW17 0RE, United Kingdom
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The Johns Hopkins Hospital, Division of pediatric allergy and immunology
Baltimore, Maryland, 21289, United States
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UT Health Houston, Department of pediatrics, division of child & adolescent neurology
Houston, Texas, 77030, United States
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University Children's Hospital Zürich - Eleonore Foundation
Zurich, CH-8008, Switzerland
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University Hospital Frankfurt, Pediatric and Adolescent Clinic
Frankfurt, 60590, Germany
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University Hospitals Birmingham NHS Foundation Trust
Birmingham, B152GW, United Kingdom
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University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
Los Angeles, California, 90095, United States
Conditions
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