Evaluating the effects of EryDex on neurological symptoms in children with Ataxia Telangiectasia
A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT)
PHASE3 · Quince Therapeutics S.p.A. · NCT06193200
This study is testing if a new treatment called EryDex can help improve neurological symptoms in children with Ataxia Telangiectasia.
Quick facts
| Phase | PHASE3 |
|---|---|
| Study type | Interventional |
| Enrollment | 106 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | Quince Therapeutics S.p.A. (industry) |
| Locations | 21 sites (Los Angeles, California and 20 other locations) |
| Trial ID | NCT06193200 on ClinicalTrials.gov |
What this trial studies
This international, multi-center, randomized, double-blind, placebo-controlled Phase 3 study aims to assess the neurological effects of EryDex, a treatment involving dexamethasone sodium phosphate infused into autologous erythrocytes, on patients diagnosed with Ataxia Telangiectasia (A-T). Participants aged 6 to 9 years will be randomized to receive either EryDex or a placebo every 28 days. The study will also include a smaller group of participants aged 10 years and older. The primary focus is to evaluate improvements in neurological symptoms associated with A-T.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 6 to 9 years with a clinical diagnosis and genetic confirmation of Ataxia Telangiectasia.
Not a fit: Patients with severe immunological impairments or those currently undergoing treatment for neoplastic diseases may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve neurological symptoms and quality of life for patients with Ataxia Telangiectasia.
How similar studies have performed: While this approach is novel in the context of A-T, similar studies using dexamethasone in other conditions have shown promising results.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Los Angeles, California and 20 other locations
- University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence — Los Angeles, California, United States (RECRUITING)
- The Johns Hopkins Hospital, Division of pediatric allergy and immunology — Baltimore, Maryland, United States (RECRUITING)
- Biotrial Inc. — Newark, New Jersey, United States (NOT_YET_RECRUITING)
- Cincinnati Children's Hospital, Division of neurology — Cincinnati, Ohio, United States (RECRUITING)
- UT Health Houston, Department of pediatrics, division of child & adolescent neurology — Houston, Texas, United States (RECRUITING)
- Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology — Copenhagen, Denmark (RECRUITING)
- Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin Kinderheilkunde III Neuropädiatrie, Allgemeinpädiatrie, Diabetologie, Endokrinologie, Sozialpädiatrie — Tübingen, Bade-Würtenberg, Germany (RECRUITING)
- University Hospital Frankfurt, Pediatric and Adolescent Clinic — Frankfurt, Germany (RECRUITING)
- IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases — Frankfurt, Germany (NOT_YET_RECRUITING)
- Spedali Civili di Brescia, Pediatric immunology department — Brescia, Italy (RECRUITING)
- Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health — Roma, Italy (RECRUITING)
- Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening — Oslo, Norway (RECRUITING)
- MedPolonia sp zoo — Poznań, Poland (NOT_YET_RECRUITING)
- Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic — Warsaw, Poland (RECRUITING)
- Hospital Universitari Vall d'Hebron, Department of pediatric neurology — Barcelona, Spain (RECRUITING)
- Hospital Universitario La Paz, Department of pediatric neurology — Madrid, Spain (RECRUITING)
- University Children's Hospital Zürich - Eleonore Foundation — Zürich, Switzerland (RECRUITING)
- University Hospitals Birmingham NHS Foundation Trust — Birmingham, United Kingdom (RECRUITING)
- St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection — London, United Kingdom (RECRUITING)
- Great Ormond Street Hospital for Children, Zayed Centre for Research — London, United Kingdom (RECRUITING)
- Nottingham Children's Hospital, Queen's Medical Center, Children's neurology — Nottingham, United Kingdom (RECRUITING)
Study contacts
- Study coordinator: Dirk Thye, MD
- Email: clinops@quincetx.com
- Phone: 415-533-3236
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Ataxia Telangiectasia, A-T, Louis-Bar syndrome, Cerebello-oculocutaneous telangiectasia