Could red blood cells deliver a steroid to ease a rare brain disorder?
NCT ID NCT06193200
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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105 people
The number who actually took part.
- Started
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Jun 2024
- Finished
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Dec 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
6 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 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 at least 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 as use of topical steroids will be permitted * A disability that may prevent the subject from completing all study requirements
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can an immune drug slow a rare childhood brain disease?
- Could early markers unlock treatments for a rare childhood disease?
- Experimental drug shows promise for rare genetic disorder
- Large study looks at how a 25-Gene cancer test affects patients and families
- Continued EryDex treatment studied in rare neurological disorder
- Precision genetic therapy aims to slow rare brain disease