Open-Label Extension of EryDex Study IEDAT-04-2022

a study on Telangiectasia

Summary

Eligibility
for people ages 6 years and up (full criteria)
Location
at UCLA
Dates
study started
completion around
Principal Investigator
by Susan L. Perlman, MD (ucla)
Headshot of Susan L. Perlman
Susan L. Perlman

Description

Summary

This is an international, multi-center, prospective, open-label, non-comparative study to provide EryDex treatment to ataxia telangiectasia (A-T) patients who complete the IEDAT-04-2022 trial on the neurological effects of EryDex on subjects with ataxia telangiectasia (NEAT trial).

Official Title

An Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)

Details

The IEDAT-05-2024 study aims to provide EryDex (dexamethasone sodium phosphate encapsulated into autologous erythrocytes) treatment to patients who complete the full study treatment period (including those receiving placebo) in the IEDAT-04-2022 (NEAT) study, who complete the study assessments, do not present safety contraindications to continuation of treatment, and provide informed consent. The open-label extension (OLE) treatment period will be 12 months. Participants will be considered to have completed the study when Visit 14 (Safety Follow-up) has been performed.

Keywords

Ataxia Telangiectasia, 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

  • body weight ≥15 kg
  • participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments

You CAN'T join if...

  • safety contraindications for continuation of treatment, as determined by the investigator
  • clinically significant immune impairment that, in the opinion of the Investigator, precludes further treatment with corticosteroids
  • Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.
  • requiring treatment with a systemic corticosteroid

Locations

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

Lead Scientist at University of California Health

  • Susan L. Perlman, MD (ucla)
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 182 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Quince Therapeutics S.p.A.
ID
NCT06664853
Phase
Phase 3 Telangiectasia Research Study
Study Type
Interventional
Participants
Expecting 106 study participants
Last Updated