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

John Bernat

Status: Active, not recruiting

Conditions: Gaucher Disease

Treatments: Biological: AVR-RD-02

Study type: Observational

Funder types: Other

Identifiers

NCT06488261

202309221

Details and patient eligibility

About

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).

Full description

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.

Enrollment: 2 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

2 participants in 1 patient group

Subjects who received AVR-RD-02

Description:

Subjects who received AVR-RD-02 in a separate parent trial

Treatment:

Biological: AVR-RD-02