Long Term Follow-Up for Safety of AVR-RD-02

N/AActive Not RecruitingNCT06488261

John Bernat2 enrolled

Overview

The goal of this observational study is to assess the safety and tolerability of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).

Subjects who received AVR-RD-02 in the preceding treatment study (NCT04145037) and who meet all eligibility criteria may participate. Subjects will be asked to return for study visits annually for 14 years to assess measures of safety and persistence of AVR-RD-02 treatment.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Gaucher Disease

Interventions

AVR-RD-02BIOLOGICAL

No study drug is administered in this study. Subjects who received AVR-RD-02 in a separate parent trial will be evaluated in this trial for long-term safety.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria Subjects must meet all of the following inclusion criteria for participation in this study: 1. Subject must be willing and able to provide written informed consent for the JAB-GD-001 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures. 2. Subject must have been enrolled in the preceding AVROBIO AVRO-RD-02-201 treatment study and have received AVR-RD-02 treatment Exclusion Criteria Subject has any medical, psychological, or other condition that, in the opinion of the Investigator: * Might interfere with the subject's participation in the study (including consenting to procedures); and/or * Poses any additional risk to the subject; and/or * Might confound the results of any study-required assessments.

Locations (1)

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Outcomes

Primary Outcomes

Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder