Evaluate the Neurological Effects of EryDex on Subjects With A-T

NCT06193200 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: IEDAT-04-2022
• Study Type: interventional
• Target: 105
• Locations: 9 countries
• Sites: 19

Brief Summary

This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate \[DSP\] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangiectasia (A-T).

Conditions

Ataxia Telangiectasia

Keywords

Ataxia Telangiectasia; A-T; Louis-Bar syndrome; Cerebello-oculocutaneous telangiectasia

Outcome Measures

Primary Outcomes (1)

• Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS)

  Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old). RmICARS (International Cooperative Ataxia Rating Scale): Score (range 0-29); higher score indicates worse disease.

  Baseline to Visit 9 (approximately 6 months)

Secondary Outcomes (2)

• Clinical Global Impression of Severity (CGI-S)

  Baseline to Visit 9 (approximately 6 months)

• Clinical Global Impression of Change (CGI-C)

  Baseline to Visit 9 (approximately 6 months)

Study Arms (2)

Dexamethasone sodium phosphate

EXPERIMENTAL

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

Drug: Dexamethasone sodium phosphate

Placebo

PLACEBO COMPARATOR

IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS)

Other: Placebo

Interventions

Dexamethasone sodium phosphate DRUG

Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion

Also known as: DSP

Dexamethasone sodium phosphate

Placebo OTHER

Placebo encapsulated in autologous erythrocytes and administered via IV infusion

Placebo

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:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

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

Study Sites (19)

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

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, 2100, Denmark

Location

University Hospital Frankfurt, Pediatric and Adolescent Clinic

Frankfurt, 60590, Germany

Location

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

Frankfurt, 60596, Germany

Location

Spedali Civili di Brescia, Pediatric immunology department

Brescia, 25123, Italy

Location

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

Roma, 00161, Italy

Location

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

Oslo, 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, 08035, Spain

Location

Hospital Universitario La Paz, Department of pediatric neurology

Madrid, 28046, Spain

Location

University Children's Hospital Zürich - Eleonore Foundation

Zurich, CH-8008, Switzerland

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, SW17 0RE, United Kingdom

Location

Great Ormond Street Hospital for Children, Zayed Centre for Research

London, WC1N 1DZ, United Kingdom

Location

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

Nottingham, NG7 2UH, 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: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2023
• First Posted: January 5, 2024
• Study Start: June 24, 2024
• Primary Completion: December 17, 2025
• Study Completion: December 17, 2025
• Last Updated: April 30, 2026
• Results First Posted: April 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

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