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

Phase 3CompletedNCT06193200

Quince Therapeutics S.p.A.105 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

105

Conditions

Ataxia Telangiectasia

Interventions

Dexamethasone sodium phosphateDRUG

Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion

PlaceboOTHER

Placebo encapsulated in autologous erythrocytes and administered via IV infusion

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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

Locations (19)

University of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence

Outcomes

Primary Outcomes

Rescored modified International Cooperative Ataxia Rating Scale (RmICARS)

Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old)

Time frame: Baseline to Visit 9 (approximately 6 months)

Secondary Outcomes

Clinical Global Impression of Severity (CGI-S)

Change in CGI-S from baseline to Visit 9

Time frame: Baseline to Visit 9 (approximately 6 months)

Clinical Global Impression of Change (CGI-C)

Clinical Global Impression of Change (CGI-C) at Visit 9

Time frame: Baseline to Visit 9 (approximately 6 months)

Data from ClinicalTrials.gov

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