To Evaluate the Effects of EryDex in Patients With A-T

Ataxia Telangiectasia Clinical Trial

— NEAT  

Official title:

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex in Subjects With Ataxia Telangiectasia (NEAT)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06193200
• Other study ID #: IEDAT-04-2022
• Status: Recruiting
• Phase: Phase 3
• Start date: June 2024
• Est. completion date: September 2025

Study information

• Verified date: June 2024
• Source: Quince Therapeutics S.p.A.
• Contact: Dirk Thye, MD
• Phone: 415-533-3236
• Email: dthye[@]quincetx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 Telangectasia (A-T).

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 information / eligibility

• Status: Recruiting
• Enrollment: 106
• Est. completion date: September 2025
• Est. primary completion date: August 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Ataxia Telangiectasia
• Cerebellar Ataxia
• Telangiectasis

Intervention

Drug:
• Dexamethasone sodium phosphate
Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion

Other:
• Placebo
Placebo encapsulated in autologous erythrocytes and administered via IV infusion

Locations

Country	Name	City	State
Belgium	UZ Leuven	Leuven
Denmark	Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology	Copenhagen
Germany	University Hospital Frankfurt, Pediatric and Adolescent Clinic	Frankfurt
Italy	Spedali Civili di Brescia, Pediatric immunology department	Brescia
Italy	Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health	Rom
Norway	Oslo University Hospital, Rikshospitalet, Division of Pediatric and Adolescent Medicine, Norwegian National Unit for Newborn Screening	Oslo
Poland	Instytut "Pomnik-Centrum Zdrowia Dziecka", Immunology clinic	Warsaw
Spain	Hospital Universitari Vall d'Hebron, Department of pediatric neurology	Barcelona
Spain	Hospital Universitario La Paz, Department of pediatric neurology	Madrid
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 States	The Johns Hopkins Hospital, Division of pediatric allergy and immunology	Baltimore	Maryland
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	University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence	Los Angeles	California

Sponsors (2)

Lead Sponsor	Collaborator
Quince Therapeutics S.p.A.	Biotrial

Countries where clinical trial is conducted

United States,  Belgium,  Denmark,  Germany,  Italy,  Norway,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rescored modified International Cooperative Ataxia Rating Scale (RmICARS)	Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old)	Baseline to Visit 9 (approximately 6 months)
Secondary	Clinical Global Impression of Severity (CGI-S)	Change in CGI-S from baseline to Visit 9	Baseline to Visit 9 (approximately 6 months)
Secondary	Clinical Global Impression of Change (CGI-C)	Clinical Global Impression of Change (CGI-C) at Visit 9	Baseline to Visit 9 (approximately 6 months)