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

Description

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).

Conditions

Ataxia Telangiectasia

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Study Overview

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Study Details

Study overview

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).

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

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

Condition
Ataxia Telangiectasia
Intervention / Treatment

-

Contacts and Locations

Los Angeles

University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence, Los Angeles, California, United States, 90095

Baltimore

The Johns Hopkins Hospital, Division of pediatric allergy and immunology, Baltimore, Maryland, United States, 21289

Hillsborough

Rare Disease Research, Hillsborough, North Carolina, United States, 27278

Cincinnati

Cincinnati Children's Hospital, Division of neurology, Cincinnati, Ohio, United States, 45229

Houston

UT Health Houston, Department of pediatrics, division of child & adolescent neurology, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

6 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Quince Therapeutics S.p.A.,

Dirk Thye, MD, STUDY_DIRECTOR, Quince Therapeutics S.p.A.

Study Record Dates

2025-09