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

Type 1 Gaucher Disease Clinical Trial

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

Status Terminated

Clinical Trial Summary

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).

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Gaucher Disease
• Type 1 Gaucher Disease

• NCT number: NCT04836377
• Study type: Observational
• Source: AVROBIO
• Status: Terminated
• Start date: July 6, 2021
• Completion date: August 21, 2023