A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

Avrobio

Status: Terminated

Conditions: Type 1 Gaucher Disease

Treatments: Other: Safety and efficacy assessments

Study type: Observational

Funder types: Industry

Identifiers

NCT04836377

AVRO-RD-02-LTF01

Details and patient eligibility

About

This is a multicenter, multinational long-term follow-up study to assess the long-term safety and durability of effect 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 and who meet all eligibility criteria may participate in the AVRO-RD-02-LTF01 study. For individual participating subjects, the Baseline visit for the AVRO-RD-02-LTF01 study will coincide with the subject's last visit in the preceding treatment study, whenever feasible. Subjects will be asked to return for study visits at approximately 6-month intervals for the first 4 years of the study and then annually thereafter for 10 years (for a total follow-up of 14 years in this study), during which time periodic safety and efficacy assessments will be performed to assess measures of safety, engraftment, and clinical response after AVR-RD-02 treatment.

Enrollment: 4 patients

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subject must have been enrolled and received AVR-RD-02 as single dose administration
• Subject must be willing and able to provide written informed consent for the AVRO-RD-02-LTF01 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures.

Exclusion criteria

• Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-02 treatment study.

Trial design

4 participants in 1 patient group

Subjects with Gaucher 1 Disease

Description:

This is a long-term follow-up study of subjects who previously received AVR-RD-02 (single dose administration) in a preceding treatment study. No investigational product will be administered in this study.

Treatment:

Other: Safety and efficacy assessments