Open-Label Extension of EryDex Study IEDAT-04-2022

NCT06664853 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: IEDAT-05-2024
• Study Type: interventional
• Target: 101
• Locations: 9 countries
• Sites: 21

Brief Summary

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).

Conditions

Ataxia Telangiectasia

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Treatment Emergent Adverse Events

  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

  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

  Number of participants presenting at least one serious adverse event (SAE)

  through study completion, up to approximately 13 months

Study Arms (1)

Dexamethasone sodium phosphate

EXPERIMENTAL

intravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)

Drug: Dexamethasone sodium phosphate

Interventions

Dexamethasone sodium phosphate DRUG

Dexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion

Dexamethasone sodium phosphate

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• body weight ≥15 kg
• participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments

You may not qualify if:

• 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

Sponsors & Collaborators

• Biotrial: collaborator
• Quince Therapeutics S.p.A.: lead

Study Sites (21)

University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence

Los Angeles, California, 90095, United States

Location

The Johns Hopkins Hospital, Division of pediatric allergy and immunology

Baltimore, Maryland, 21289, United States

Location

Saint Peter's University Hospital, Inc.

New Brunswick, New Jersey, 08901, United States

Location

Cincinnati Children's Hospital, Division of neurology

Cincinnati, Ohio, 45229, United States

Location

UT Health Houston, Department of pediatrics, division of child & adolescent neurology

Houston, Texas, 77030, United States

Location

Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology

Copenhagen, Denmark, 2100, Denmark

Location

University Hospital Frankfurt, Pediatric and Adolescent Clinic

Frankfurt, Germany, 60590, Germany

Location

IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases

Frankfurt am Main, Hesse, 60596, Germany

Location

Spedali Civili di Brescia, Pediatric immunology department

Brescia, Italy, 25213, Italy

Location

Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health

Roma, Italy, 00161, Italy

Location

Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening

Oslo, Norway, 0372, Norway

Location

MedPolonia sp zoo

Poznan, 60-693, Poland

Location

Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic

Warsaw, 04-736, Poland

Location

Hospital Universitari Vall d'Hebron, Department of pediatric neurology

Barcelona, Spain, 08035, Spain

Location

Hospital Universitario La Paz, Department of pediatric neurology

Madrid, Spain, 28046, Spain

Location

Universitary Hospital Virgen del Rocío

Seville, 41013, Spain

Location

University Children's Hospital Zürich - Eleonore Foundation

Zurich, CH 8008, Switzerland

Location

Great Ormond Street Hospital for Children, Zayed Centre for Research

London, United Kingdom, WC1N 1DZ, United Kingdom

Location

Nottingham Children's Hospital, Queen's Medical Center, Children's neurology

Nottingham, United Kingdom, NG7 2UH, United Kingdom

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, B152GW, United Kingdom

Location

St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection

London, SE1 7EH, United Kingdom

Location

MeSH Terms

Conditions

Ataxia Telangiectasia

Interventions

dexamethasone 21-phosphate

Condition Hierarchy (Ancestors)

Spinocerebellar Ataxias; Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocutaneous Syndromes; Ataxia; Dyskinesias; Neurologic Manifestations; Telangiectasis; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Primary Immunodeficiency Diseases; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Immunologic Deficiency Syndromes; Immune System Diseases

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Quince Therapeutics

clinops@quincetx.com

+1 415 910 5717

Study Officials

• Dirk Thye, MD

  Quince Therapeutics S.p.A.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 25, 2024
• First Posted: October 30, 2024
• Study Start: December 11, 2024
• Primary Completion: January 30, 2026
• Study Completion: January 30, 2026
• Last Updated: May 5, 2026
• Results First Posted: May 5, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

DE (2); ES (3); US (5); CH (1); DK (1); NO (1); GB (4); IT (2); PL (2)