Evaluate the Neurological Effects of EryDex on Subjects With A-T
- Conditions
- Ataxia Telangiectasia
- Interventions
- Other: Placebo
- Registration Number
- NCT06193200
- Lead Sponsor
- Quince Therapeutics S.p.A.
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 106
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Dexamethasone sodium phosphate Dexamethasone sodium phosphate IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) Placebo Placebo IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS)
- Primary Outcome Measures
Name Time Method Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) Baseline to Visit 9 (approximately 6 months) Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old)
- Secondary Outcome Measures
Name Time Method Clinical Global Impression of Change (CGI-C) Baseline to Visit 9 (approximately 6 months) Clinical Global Impression of Change (CGI-C) at Visit 9
Clinical Global Impression of Severity (CGI-S) Baseline to Visit 9 (approximately 6 months) Change in CGI-S from baseline to Visit 9
Trial Locations
- Locations (21)
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
Biotrial Inc.
🇺🇸Newark, New Jersey, 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
Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin Kinderheilkunde III Neuropädiatrie, Allgemeinpädiatrie, Diabetologie, Endokrinologie, Sozialpädiatrie
🇩🇪Tübingen, Bade-Würtenberg, Germany
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
🇵🇱Poznań, 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
🇨🇭Zürich, 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