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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
101
Dexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion
Number of Participants With Treatment Emergent Adverse Events
Number of participants presenting at least one treatment emergent adverse event (TEAE)
Time frame: through study completion, up to approximately 13 months
Number of Participants With Treatment Emergent Adverse Events Leading to Intervention Discontinuation
Number of participants presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation
Time frame: through study completion, up to approximately 13 months
Number of Participants With Serious Adverse Events
Number of participants presenting at least one serious adverse event (SAE)
Time frame: through study completion, up to approximately 13 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
Los Angeles, California, United States
The Johns Hopkins Hospital, Division of pediatric allergy and immunology
Baltimore, Maryland, United States
Saint Peter's University Hospital, Inc.
New Brunswick, New Jersey, United States
Cincinnati Children's Hospital, Division of neurology
Cincinnati, Ohio, United States
UT Health Houston, Department of pediatrics, division of child & adolescent neurology
Houston, Texas, United States
Copenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
Copenhagen, Denmark, Denmark
University Hospital Frankfurt, Pediatric and Adolescent Clinic
Frankfurt, Germany, Germany
IKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
Frankfurt am Main, Hesse, Germany
Spedali Civili di Brescia, Pediatric immunology department
Brescia, Italy, Italy
Policlinico Umberto I, La sapienza University, Department of neurosciences and menthal health
Roma, Italy, Italy
...and 11 more locations