Clinical trial NCT06193200: Evaluate the Neurological Effects of EryDex on Subjects With A-T
Official title: A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Ataxia Telangiectasia
Keywords: Ataxia Telangiectasia
A-T
Louis-Bar syndrome
Cerebello-oculocutaneous telangiectasia
Lead sponsor: Quince Therapeutics S.p.A.
Sponsor class: INDUSTRY
Collaborators: name: Biotrial
First posted: 2024-01-05
Enrollment: 106
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Rescored modified International Cooperative Ataxia Rating Scale (RmICARS)
timeFrame: Baseline to Visit 9 (approximately 6 months)
Secondary outcomes:
measure: Clinical Global Impression of Severity (CGI-S)
timeFrame: Baseline to Visit 9 (approximately 6 months)
measure: Clinical Global Impression of Change (CGI-C)
timeFrame: Baseline to Visit 9 (approximately 6 months)
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 & adolescent neurology
state: Texas
city: Copenhagen
country: Denmark
facility: Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
city: Tübingen
country: Germany
facility: Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin Kinderheilkunde III Neuropädiatrie, Allgemeinpädiatrie, Diabetologie, Endokrinologie, Sozialpädiatrie
state: Bade-Würtenberg
city: Frankfurt
country: Germany
facility: University Hospital Frankfurt, Pediatric and Adolescent Clinic
city: Frankfurt
country: Germany
facility: IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
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: Poznań
country: Poland
facility: MedPolonia sp zoo
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: