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).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
105
Dexamethasone Sodium Phosphate encapsulated in autologous erythrocytes and administered via intravenous (IV) infusion
Incidence of treatment emergent adverse events
Number of patients presenting at least one treatment emergent adverse event (TEAE)
Time frame: through study completion, up to 14 months
Incidence of treatment emergent adverse events leading to intervention discontinuation
Number of patients presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation
Time frame: through study completion, up to 14 months
Incidence of serious adverse events
Number of patients presenting at least one serious adverse event (SAE)
Time frame: through study completion, up to 14 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
ACTIVE_NOT_RECRUITINGThe Johns Hopkins Hospital, Division of pediatric allergy and immunology
Baltimore, Maryland, United States
ACTIVE_NOT_RECRUITINGSaint Peter's University Hospital, Inc.
New Brunswick, New Jersey, United States
NOT_YET_RECRUITINGBiotrial Inc.
Newark, New Jersey, United States
WITHDRAWNCincinnati Children's Hospital, Division of neurology
Cincinnati, Ohio, United States
TERMINATEDUT Health Houston, Department of pediatrics, division of child & adolescent neurology
Houston, Texas, United States
ACTIVE_NOT_RECRUITINGCopenhagen University Hospital, Rigshospitalet, Department of Pediatric Neurology
Copenhagen, Denmark
ACTIVE_NOT_RECRUITINGIKF Pneumologie GmbH & Co. KG; Institut für klinische Forschung Pneumologie Clinical Research Center Respiratory Diseases
Frankfurt am Main, Hesse, Germany
ACTIVE_NOT_RECRUITINGUniversity Hospital Frankfurt, Pediatric and Adolescent Clinic
Frankfurt, Germany
ACTIVE_NOT_RECRUITINGSpedali Civili di Brescia, Pediatric immunology department
Brescia, Italy
ACTIVE_NOT_RECRUITING...and 12 more locations