Open-Label Extension of EryDex Study IEDAT-04-2022
- 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
- body weight ≥15 kg
- participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Dexamethasone sodium phosphate Dexamethasone sodium phosphate intravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)
- Primary Outcome Measures
Name Time Method Incidence of treatment emergent adverse events through 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 discontinuation through 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 events through study completion, up to 14 months Number of patients presenting at least one serious adverse event (SAE)
- Secondary Outcome Measures
Name Time Method
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