NCT06193200

Evaluate the Neurological Effects of EryDex on Subjects With A-T

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 105 (actual)
• Sponsor: Quince Therapeutics S.p.A. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Ataxia Telangiectasia

Interventions

Type	Name	Description
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

Timeline

• Start date: 2024-06-24
• Primary completion: 2025-12-17
• Completion: 2025-12-17
• First posted: 2024-01-05
• Last updated: 2026-01-13

Locations

19 sites across 9 countries: United States, Denmark, Germany, Italy, Norway, Poland, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study