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

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

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

The IEDAT-05-2024 study aims to provide EryDex (dexamethasone sodium phosphate encapsulated into autologous erythrocytes) treatment to all participants in the IEDAT-04-2022 (NEAT) study, who complete the study assessments, do not -have safety contraindications to continuation of treatment, and who 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.

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