Overview
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).
Description
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.
Eligibility
Inclusion Criteria:
- body weight ≥15 kg
- participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments
Exclusion Criteria:
- 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