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 Telangiectasia (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

ArmDescriptionAssigned Intervention
Experimental
Dexamethasone sodium phosphate 		IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)
  • Drug: Dexamethasone sodium phosphate
    Dexamethasone sodium phosphate encapsulated in autologous erythrocytes and administered via IV infusion
    Other names:
    • DSP
Placebo Comparator
Placebo 		IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS)
  • Other: Placebo
    Placebo encapsulated in autologous erythrocytes and administered via IV infusion

Recruiting Locations

UT Health Houston, Department of pediatrics, division of child & adolescent neurology
Houston, Texas 77030
Contact:
Sam Nicholas Russo, MD

More Details

Status
Recruiting
Sponsor
Quince Therapeutics S.p.A.

Study Contact

Dirk Thye, MD
415-533-3236
clinops@quincetx.com

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.