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

About

Brief 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).

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase 3

Eligibility

Gender

All

Healthy Volunteers

No

Minimum Age

6 Years

Maximum Age

N/A

Inclusion 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

Exclusion Criteria:

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