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

Phase 3
Recruiting
Conditions
Ataxia Telangiectasia
Interventions
Registration Number
NCT06664853
Lead Sponsor
Quince Therapeutics S.p.A.
Brief Summary

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

Detailed Description

The IEDAT-05-2024 study aims to provide EryDex (dexamethasone sodium phosphate encapsulated into autologous erythrocytes) treatment to patients who complete the full study treatment period (including those receiving placebo) in the IEDAT-04-2022 (NEAT) study, who complete the study assessments, do not present safety contraindications to continuation of treatment, and 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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
106
Inclusion Criteria
  • body weight ≥15 kg
  • participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments
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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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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Dexamethasone sodium phosphateDexamethasone sodium phosphateintravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)
Primary Outcome Measures
NameTimeMethod
Incidence of treatment emergent adverse eventsthrough study completion, up to 14 months

Number of patients presenting at least one treatment emergent adverse event (TEAE)

Incidence of treatment emergent adverse events leading to intervention discontinuationthrough study completion, up to 14 months

Number of patients presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation

Incidence of serious adverse eventsthrough study completion, up to 14 months

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

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (17)

University Hospital Frankfurt, Pediatric and Adolescent Clinic

🇩🇪

Frankfurt, Germany

Spedali Civili di Brescia, Pediatric immunology department

🇮🇹

Brescia, Italy

Tübingen University Hospital, Division Translational Genomics of Neurodegenerative Diseases

🇩🇪

Tübingen, Bade-Würtenberg, Germany

University Hospitals Birmingham NHS Foundation Trust

🇬🇧

Birmingham, United Kingdom

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

UZ Leuven

🇧🇪

Leuven, Belgium

Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology

🇩🇰

Copenhagen, Denmark

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

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

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

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