Continued EryDex treatment studied in rare neurological disorder

NCT ID NCT06664853

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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

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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

  • 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

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