Continued EryDex treatment studied in rare neurological disorder
NCT ID NCT06664853
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study offered continued treatment with EryDex to 101 people with ataxia telangiectasia (A-T) who had finished a previous trial. The main goal was to monitor safety, including side effects and serious events. The study was terminated early, and it did not aim to cure the disease but to manage its symptoms.
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
-
Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
-
101 people
The number who actually took part.
- Started
-
Dec 2024
- Finished
-
Jan 2026
- Lead sponsor
-
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
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * body weight ≥15 kg * participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments Exclusion Criteria: * safety contraindications for continuation of treatment, as determined by the investigator * clinically significant immune impairment that, in the opinion of the Investigator, precludes further treatment with corticosteroids * Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years. * requiring treatment with a systemic corticosteroid
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Ataxia telangiectasia are added.
By submitting, you agree to our Terms of use
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
-
Cincinnati Children's Hospital, Division of neurology
Cincinnati, Ohio, 45229, United States
-
Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
Copenhagen, Denmark, 2100, Denmark
-
Great Ormond Street Hospital for Children, Zayed Centre for Research
London, United Kingdom, WC1N 1DZ, United Kingdom
-
Hospital Universitari Vall d'Hebron, Department of pediatric neurology
Barcelona, Spain, 08035, Spain
-
Hospital Universitario La Paz, Department of pediatric neurology
Madrid, Spain, 28046, Spain
-
IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
Frankfurt am Main, Hesse, 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, United Kingdom, NG7 2UH, United Kingdom
-
Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening
Oslo, Norway, 0372, Norway
-
Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
Roma, Italy, 00161, Italy
-
Saint Peter's University Hospital, Inc.
New Brunswick, New Jersey, 08901, United States
-
Spedali Civili di Brescia, Pediatric immunology department
Brescia, Italy, 25213, Italy
-
St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection
London, SE1 7EH, 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
-
Universitary Hospital Virgen del Rocío
Seville, 41013, Spain
-
University Children's Hospital Zürich - Eleonore Foundation
Zurich, CH 8008, Switzerland
-
University Hospital Frankfurt, Pediatric and Adolescent Clinic
Frankfurt, Germany, 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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- 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
- Could red blood cells deliver a steroid to ease a rare brain disorder?
- Precision genetic therapy aims to slow rare brain disease
- New study hopes to outsmart pancreatic cancer by gathering clues