Open
Actively Recruiting
To Evaluate the Effects of EryDex in Patients 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 Telangectasia (A-T).
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
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
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Study Stats
Protocol No.
23-001891
Category
Brain/Neurological Diseases
Genetic and Rare Diseases
Pediatric and Prenatal Disorders
Principal Investigator
Contact
Location
- UCLA Westwood