MedTrace Logo

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

NCT04999059 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 5

Last updated 2023-08-31

No results posted yet for this study

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.

Conditions

Interventions

OTHER

Safety and Efficacy Assessments

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

Sponsors & Collaborators

  • AVROBIO

    lead INDUSTRY

Principal Investigators

  • Inderpal Panesar, MRPharmS · AVROBIO, Inc

Eligibility

Min Age
16 Years
Max Age
50 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-05-08
Primary Completion
2023-08-16
Completion
2023-08-16
FDA Drug
Yes

Countries

  • Australia
  • Brazil

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04999059 on ClinicalTrials.gov