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Evaluate the Neurological Effects of EryDex on Subjects With A-T

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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
Treatment
Study Type
Interventional
Phase
Phase 3

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
6 Years
Maximum Age
N/A

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
Contact
Dillan Hayward
Location
  • UCLA Westwood
For Providers
NCT No.
NCT06193200
For detailed technical eligibility, visit ClinicalTrials.gov.