Evaluate the Neurological Effects of EryDex on Subjects With A-T (NCT06193200) | Clinical Trial Compass
CompletedPhase 3
Evaluate the Neurological Effects of EryDex on Subjects With A-T
United States, Denmark, Germany105 participantsStarted 2024-06-24
Plain-language 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).
Who can participate
Age range
6 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS)
Timeframe: Baseline to Visit 9 (approximately 6 months)