Phase 3
Completed N=105
Evaluate the Neurological Effects of EryDex on Subjects With A-T
Source: ClinicalTrials.gov NCT06193200 ↗Enrolled (actual)
105
Serious AEs
4.8%
Results posted
Apr 2026
Primary outcomePrimary: Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) — 0.94; 2.24 RmICARS score (0-29 points)
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
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 Telangiectasia (A-T).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) |
0.94; 2.24 | — |
| SECONDARY Clinical Global Impression of Severity (CGI-S) |
8; 7 | — |
| SECONDARY Clinical Global Impression of Change (CGI-C) |
13; 6 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT06193200). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.