Clinical trial NCT06664853: Open-Label Extension of EryDex Study IEDAT-04-2022
Official title: An Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Ataxia Telangiectasia
Lead sponsor: Quince Therapeutics S.p.A.
Sponsor class: INDUSTRY
Collaborators: name: Biotrial
First posted: 2024-10-30
Enrollment: 106
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Incidence of treatment emergent adverse events
timeFrame: through study completion, up to 14 months
measure: Incidence of treatment emergent adverse events leading to intervention discontinuation
timeFrame: through study completion, up to 14 months
measure: Incidence of serious adverse events
timeFrame: through study completion, up to 14 months
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
state: California
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital, Division of pediatric allergy and immunology
state: Maryland
city: Newark
country: United States
facility: Biotrial Inc.
state: New Jersey
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital, Division of neurology
state: Ohio
city: Houston
country: United States
facility: UT Health Houston, Department of pediatrics, division of child &amp;amp; adolescent neurology
state: Texas
city: Copenhagen
country: Denmark
facility: Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
city: Tübingen
country: Germany
facility: Tübingen University Hospital, Division Translational Genomics of Neurodegenerative Diseases
state: Bade-Würtenberg
city: Frankfurt
country: Germany
facility: University Hospital Frankfurt, Pediatric and Adolescent Clinic
city: Brescia
country: Italy
facility: Spedali Civili di Brescia, Pediatric immunology department
city: Roma
country: Italy
facility: Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
city: Oslo
country: Norway
facility: Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening
city: Warsaw
country: Poland
facility: Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron, Department of pediatric neurology
city: Madrid
country: Spain
facility: Hospital Universitario La Paz, Department of pediatric neurology
city: Zürich
country: Switzerland
facility: University Children's Hospital Zürich - Eleonore Foundation
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: London
country: United Kingdom
facility: St George's University Hospitals NHS Foundation Trust, Centre for Neonatal and Paediatric Infection
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children, Zayed Centre for Research
city: Nottingham
country: United Kingdom
facility: Nottingham Children's Hospital, Queen's Medical Center, Children's neurology
Study officials:
affiliation: Quince Therapeutics S.p.A.
name: Dirk Thye, MD
role: STUDY_DIRECTOR
References: