NCT04836377

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

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 4 (actual)
• Sponsor: AVROBIO · Industry
• Sex: All
• Age: 18 Years – 99 Years
• Healthy volunteers: Not accepted

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).

Detailed 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.

Conditions

• Type 1 Gaucher Disease

Interventions

Type	Name	Description
OTHER	Safety and efficacy assessments	Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention

Timeline

• Start date: 2021-07-06
• Primary completion: 2023-08-21
• Completion: 2023-08-21
• First posted: 2021-04-08
• Last updated: 2023-08-29

Locations

3 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study