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Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study

NCT03563053 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 104

Last updated 2024-10-08

Study results available
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Summary

Primary Objective

To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.

Secondary Objective

To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).

Exploratory Objective:

To evaluate the long-term effect of EDS-EP in treating central nervous system (CNS) symptoms, as measured by the "Modified" International Cooperative Ataxia Rating Scale (mICARS), and Clinical Global Impression of severity and change (CGI-S/C).

Conditions

  • Ataxia Telangiectasia
  • Genetic Syndrome

Interventions

COMBINATION_PRODUCT

EryDex System

EryDex System was a combination product that was used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EDS) creating the EDS-end product which was infused into the patients. EryDex treatment consisted of a dose range correspondent to the ATTeST High Dose (obtained by loading 125 mg of DSP to the EryDex process and that, in the ATTeST, resulted in a mean of 17.4 ± 5.4 mg (mean ± standard deviation) of DSP infused to patients) administered via ex vivo encapsulation into EDS that were infused into the patient with A-T. The dose of EryDex treatment was selected to allow collection of long-term safety data on the dose of erythrocyte encapsulated DSP that was considered more effective among the two different doses that were employed in the ATTeST study, based on the analysis of the ATTeST blinded, Phase 3 study data.

Sponsors & Collaborators

  • Quince Therapeutics S.p.A.

    lead INDUSTRY

Principal Investigators

  • Guenter R Janhofer, MD, PhD · EryDel S.p.A.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-06-12
Primary Completion
2022-09-02
Completion
2022-09-02
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Germany
  • India
  • Italy
  • Norway
  • Poland
  • Spain
  • Tunisia
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03563053 on ClinicalTrials.gov