Evaluate the Neurological Effects of EryDex on Subjects With A-T
Purpose
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).
Condition
- Ataxia Telangiectasia
Eligibility
- Eligible Ages
- Over 6 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Dexamethasone sodium phosphate |
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS) |
|
|
Placebo Comparator Placebo |
IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS) |
|
Recruiting Locations
Houston, Texas 77030
Sam Nicholas Russo, MD
More Details
- Status
- Recruiting
- Sponsor
- Quince Therapeutics S.p.A.
Detailed Description
The EryDex System (EDS) is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) which is infused into the patient. In the placebo arm, the subjects will receive autologous erythrocytes prepared with the EDS process using a placebo solution. Upon completion of all screening assessments for eligibility, subjects meeting all selection criteria at baseline will be randomized in a 1:1 fashion to EryDex or placebo. Approximately 86 subjects 6- to 9-years-old, approximately 43 per group, will be randomized. Approximately 20 subjects 10 years of age and above, 10 per treatment group, may also be enrolled.