To Evaluate the Effects of EryDex in Patients With A-T

• Status: Recruiting
• Trial end date: 2025-09-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Quince Therapeutics S.p.A.
• Collaborator: Biotrial
• Treatments: BB 1101; Dexamethasone; Dexamethasone 21-phosphate; Dexamethasone acetate

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