Providing EryDex treatment to patients with ataxia telangiectasia
An Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)
This study is testing if a new treatment called EryDex can be safe and effective for people with ataxia telangiectasia who have already participated in a previous trial.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 106 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | Quince Therapeutics S.p.A. Industry-sponsored |
| Locations | 19 sites (Los Angeles, California and 18 other locations) |
| Trial ID | NCT06664853 on ClinicalTrials.gov |
What this trial studies
This international, multi-center, open-label study offers EryDex treatment to patients with ataxia telangiectasia who have completed the IEDAT-04-2022 trial. The study aims to assess the safety and efficacy of EryDex, which is dexamethasone sodium phosphate encapsulated into autologous erythrocytes, over a 12-month treatment period. Participants must have completed the previous study and meet specific safety criteria to continue treatment. The study will monitor participants for any adverse effects and overall treatment outcomes.
Who should consider this trial
Good fit: Ideal candidates are patients with ataxia telangiectasia who have completed the IEDAT-04-2022 study and meet the inclusion criteria.
Not a fit: Patients with significant immune impairment or contraindications to corticosteroid treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve neurological outcomes for patients with ataxia telangiectasia.
How similar studies have performed: Other studies involving corticosteroid treatments for neurological conditions have shown promise, but this specific approach is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * body weight ≥15 kg * participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments 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
Where this trial is running
Los Angeles, California and 18 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 (Not_yet_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 (Not_yet_recruiting)
- Tübingen University Hospital, Division Translational Genomics of Neurodegenerative Diseases — Tübingen, Bade-Würtenberg, Germany (Not_yet_recruiting)
- University Hospital Frankfurt, Pediatric and Adolescent Clinic — Frankfurt, Germany (Recruiting)
- Spedali Civili di Brescia, Pediatric immunology department — Brescia, Italy (Not_yet_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)
- Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic — Warsaw, Poland (Recruiting)
- Hospital Universitari Vall d'Hebron, Department of pediatric neurology — Barcelona, Spain (Not_yet_recruiting)
- Hospital Universitario La Paz, Department of pediatric neurology — Madrid, Spain (Recruiting)
- University Children's Hospital Zürich - Eleonore Foundation — Zürich, Switzerland (Not_yet_recruiting)
- University Hospitals Birmingham NHS Foundation Trust — Birmingham, United Kingdom (Not_yet_recruiting)
- St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection — London, United Kingdom (Not_yet_recruiting)
- Great Ormond Street Hospital for Children, Zayed Centre for Research — London, United Kingdom (Not_yet_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.