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

NCT ID: NCT06193200

Last Updated: 2026-01-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 105 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-06-24
• Study Completion Date: 2025-12-17

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).

Detailed Description

The EryDex System (EDS) is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) which is infused into the patient.

In the placebo arm, the subjects will receive autologous erythrocytes prepared with the EDS process using a placebo solution.

Upon completion of all screening assessments for eligibility, subjects meeting all selection criteria at baseline will be randomized in a 1:1 fashion to EryDex or placebo. Approximately 86 subjects 6- to 9-years-old, approximately 43 per group, will be randomized. Approximately 20 subjects 10 years of age and above, 10 per treatment group, may also be enrolled.

Conditions

Ataxia Telangiectasia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Dexamethasone sodium phosphate

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

Group Type: EXPERIMENTAL

Dexamethasone sodium phosphate

Intervention Type: DRUG

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

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Placebo encapsulated in autologous erythrocytes and administered via IV infusion

Interventions

Dexamethasone sodium phosphate

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

Intervention Type: DRUG

Placebo

Placebo encapsulated in autologous erythrocytes and administered via IV infusion

Intervention Type: OTHER

Other Intervention Names

DSP

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Biotrial

INDUSTRY

Sponsor Role: collaborator

Quince Therapeutics S.p.A.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Dirk Thye, MD

Role: STUDY_DIRECTOR

Quince Therapeutics S.p.A.

Locations

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

Los Angeles, California, United States

The Johns Hopkins Hospital, Division of pediatric allergy and immunology

Baltimore, Maryland, United States

Cincinnati Children's Hospital, Division of neurology

Cincinnati, Ohio, United States

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

Houston, Texas, United States

Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology

Copenhagen, , Denmark

University Hospital Frankfurt, Pediatric and Adolescent Clinic

Frankfurt, , Germany

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

Frankfurt, , Germany

Spedali Civili di Brescia, Pediatric immunology department

Brescia, , Italy

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

Roma, , Italy

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

Oslo, , Norway

MedPolonia sp zoo

Poznan, , Poland

Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic

Warsaw, , Poland

Hospital Universitari Vall d'Hebron, Department of pediatric neurology

Barcelona, , Spain

Hospital Universitario La Paz, Department of pediatric neurology

Madrid, , Spain

University Children's Hospital Zürich - Eleonore Foundation

Zurich, , Switzerland

University Hospitals Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

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

London, , United Kingdom

Great Ormond Street Hospital for Children, Zayed Centre for Research

London, , United Kingdom

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

Nottingham, , United Kingdom

Countries

United States; Denmark; Germany; Italy; Norway; Poland; Spain; Switzerland; United Kingdom

Other Identifiers

IEDAT-04-2022

Identifier Type: -

Identifier Source: org_study_id