This study is in progress, not accepting new patients

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

a study on Telangiectasia Cerebellar Ataxia

Summary

Eligibility
for people ages 6 years and up (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
study ends around
Principal Investigator
by Susan L. Perlman, MD
Headshot of Susan L. Perlman
Susan L. Perlman

Description

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).

Official Title

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

Details

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.

Keywords

Ataxia Telangiectasia, A-T, Louis-Bar syndrome, Cerebello-oculocutaneous telangiectasia, Ataxia, Cerebellar Ataxia, Telangiectasis, Dexamethasone, Dexamethasone acetate, Dexamethasone 21-phosphate, BB 1101, Dexamethasone sodium phosphate

Eligibility

You can join if…

Open to people ages 6 years and up

  • 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

You CAN'T join if...

  • 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

Locations

  • University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
    Los Angeles California 90095 United States
  • UT Health Houston, Department of pediatrics, division of child & adolescent neurology
    Houston Texas 77030 United States

Lead Scientist at UCLA

  • Susan L. Perlman, MD
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 191 research publications

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Quince Therapeutics S.p.A.
Links
Sign up for this study
ID
NCT06193200
Phase
Phase 3 research study
Study Type
Interventional
Participants
About 105 people participating
Last Updated