Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201

NCT ID: NCT04999059

Last Updated: 2023-08-31

Basic Information

• Recruitment Status: TERMINATED
• Total Enrollment: 5 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-05-08
• Study Completion Date: 2023-08-16

Brief Summary

This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201 (treatment study). No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from AVR-RD-01 gene therapy infusion.

Detailed Description

Participants enrolled in the AVRO-RD-01-201 study will be offered participation in the AVRO-RD-01-LTF01 study. The Baseline visit for the AVRO-RD-01-LTF01 study will coincide with the participant's last visit in the AVRO-RD-01-201 study. Participants confirmed eligible for the AVRO-RD-01-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for 10 years (for a total of 15 years from AVR-RD-01 infusion) during which time continued safety, engraftment, and efficacy of treatment will be assessed.

Conditions

Fabry Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Participants with Fabry Disease

This is a long-term follow-up study of participants who previously received AVR-RD-01 (single dose administration) in the AVRO-RD-01-201 treatment study. No investigational product will be administered in this study.

Safety and Efficacy Assessments

Intervention Type: OTHER

Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.

Interventions

Safety and Efficacy Assessments

Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.

Intervention Type: OTHER

Other Intervention Names

Gene Therapy Intervention

Eligibility Criteria

Inclusion Criteria

1. Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study.

Exclusion Criteria

1. 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.

2. Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-01 treatment study.

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

AVROBIO

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Inderpal Panesar, MRPharmS

Role: STUDY_DIRECTOR

AVROBIO, Inc

Locations

Royal Melbourne Hospital

Melbourne, Parkville VIC, Australia

Royal Perth Hospital

Perth, , Australia

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Countries

Australia; Brazil

Other Identifiers

AVRO-RD-01-LTF01

Identifier Type: -

Identifier Source: org_study_id