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

Avrobio

Status: Terminated

Conditions: Fabry Disease

Treatments: Other: Safety and Efficacy Assessments

Study type: Observational

Funder types: Industry

Identifiers

NCT04999059

AVRO-RD-01-LTF01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 5 patients

Sex: Male

Ages: 16 to 50 years old

Volunteers: No Healthy Volunteers

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.

Trial design

5 participants in 1 patient group

Participants with Fabry Disease

Description:

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.

Treatment:

Other: Safety and Efficacy Assessments