Open-Label Extension of EryDex Study IEDAT-04-2022 (OLE_NEAT)

April 15, 2026 updated by: Quince Therapeutics S.p.A.

An Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)

This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied the neurological effects of EryDex on subjects with ataxia telangiectasia (NEAT trial).

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

The IEDAT-05-2024 study aims to provide EryDex (dexamethasone sodium phosphate encapsulated into autologous erythrocytes) treatment to all participants in the IEDAT-04-2022 (NEAT) study, who complete the study assessments, do not -have safety contraindications to continuation of treatment, and who provide informed consent. The open-label extension (OLE) treatment period will be 12 months. Participants will be considered to have completed the study when Visit 14 (Safety Follow-up) has been performed.

Study Type

Interventional

Enrollment (Actual)

101

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Denmark
    • Denmark
      • Copenhagen, Denmark, Denmark, 2100
        • Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
  • Germany
    • Germany
      • Frankfurt, Germany, Germany, 60590
        • University Hospital Frankfurt, Pediatric and Adolescent Clinic
    • Hesse
      • Frankfurt am Main, Hesse, Germany, 60596
        • IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
  • Italy
    • Italy
      • Brescia, Italy, Italy, 25213
        • Spedali Civili di Brescia, Pediatric immunology department
      • Roma, Italy, Italy, 00161
        • Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
  • Norway
    • Norway
      • Oslo, Norway, Norway, 0372
        • Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening
  • Poland
      • Poznan, Poland, 60-693
        • MedPolonia sp zoo
      • Warsaw, Poland, 04-736
        • Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic
  • Spain
      • Seville, Spain, 41013
        • Universitary Hospital Virgen del Rocio
    • Spain
      • Barcelona, Spain, Spain, 08035
        • Hospital Universitari Vall d'Hebron, Department of pediatric neurology
      • Madrid, Spain, Spain, 28046
        • Hospital Universitario La Paz, Department of pediatric neurology
  • Switzerland
      • Zurich, Switzerland, CH 8008
        • University Children's Hospital Zürich - Eleonore Foundation
  • United Kingdom
      • Birmingham, United Kingdom, B152GW
        • University Hospitals Birmingham NHS Foundation Trust
      • London, United Kingdom, SE1 7EH
        • St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection
    • United Kingdom
      • London, United Kingdom, United Kingdom, WC1N 1DZ
        • Great Ormond Street Hospital for Children, Zayed Centre for Research
      • Nottingham, United Kingdom, United Kingdom, NG7 2UH
        • Nottingham Children's Hospital, Queen's Medical Center, Children's neurology
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
    • Maryland
      • Baltimore, Maryland, United States, 21289
        • The Johns Hopkins Hospital, Division of pediatric allergy and immunology
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Saint Peter's University Hospital, Inc.
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital, Division of neurology
    • Texas
      • Houston, Texas, United States, 77030
        • UT Health Houston, Department of pediatrics, division of child & adolescent neurology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dexamethasone sodium phosphate
intravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)
Drug: Dexamethasone sodium phosphate
Dexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment Emergent Adverse Events
Time Frame: through study completion, up to approximately 13 months
Number of participants presenting at least one treatment emergent adverse event (TEAE)
through study completion, up to approximately 13 months
Number of Participants With Treatment Emergent Adverse Events Leading to Intervention Discontinuation
Time Frame: through study completion, up to approximately 13 months
Number of participants presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation
through study completion, up to approximately 13 months
Number of Participants With Serious Adverse Events
Time Frame: through study completion, up to approximately 13 months
Number of participants presenting at least one serious adverse event (SAE)
through study completion, up to approximately 13 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Quince Therapeutics S.p.A.

Collaborators

Biotrial

Investigators

  • Study Director: Dirk Thye, MD, Quince Therapeutics S.p.A.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 11, 2024

Primary Completion (Actual)

January 30, 2026

Study Completion (Actual)

January 30, 2026

Study Registration Dates

First Submitted

October 25, 2024

First Submitted That Met QC Criteria

October 28, 2024

First Posted (Actual)

October 30, 2024

Study Record Updates

Last Update Posted (Actual)

May 5, 2026

Last Update Submitted That Met QC Criteria

April 15, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IEDAT-05-2024

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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