Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
- Conditions
- Fabry Disease
- Registration Number
- NCT04999059
- Lead Sponsor
- AVROBIO
- 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 5
- Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study.
-
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.
-
Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-01 treatment study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Presence of anti-Alpha-galactosidase A (AGA) antibodies Baseline to Year 15 post gene therapy infusion Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests Baseline to Year 15 post gene therapy infusion Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA) Baseline to Year 15 post gene therapy infusion Incidence of clinically significant AEs and SAEs Baseline to Year 15 post gene therapy infusion Presence of replication competent lentivirus (RCL) Baseline to Year 15 post gene therapy infusion Number of participants with clinically relevant abnormalities, as assessed by vital signs Baseline to Year 15 post gene therapy infusion
- Secondary Outcome Measures
Name Time Method Change from baseline in eGFR Baseline to Year 15 post gene therapy infusion Change from baseline in AGA enzyme activity level and peripheral blood leukocytes (PBLs) Baseline to Year 15 post gene therapy infusion Change from baseline in abdominal pain and stool consistency as assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D) Baseline to Year 15 post gene therapy infusion Change from baseline in Globotriaosylceramide (Gb3) biomarkers for Fabry disease in plasma and urine Baseline to Year 15 post gene therapy infusion Average Vector Copy Number (VCN) in bone marrow / progenitor cells as assessed by quantitative polymerase chain reaction (qPCR) and/or droplet digital polymerase chain reaction (ddPCR) Baseline to Year 15 post gene therapy infusion Change from baseline in left ventricular mass index (LVMI) as assessed by cardiac magnetic resonance imaging (MRI) Baseline to Year 15 post gene therapy infusion Change from baseline in Brief Pain Inventory-Short Form (BPI-SF) questionnaire scores Baseline to Year 15 post gene therapy infusion Change from baseline in physical and mental functioning as assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) scores Baseline to Year 15 post gene therapy infusion Average Vector Copy Number (VCN) in peripheral blood leukocytes as assessed by quantitative polymerase chain reaction (qPCR) and/or droplet digital polymerase chain reaction (ddPCR) Baseline to Year 15 post gene therapy infusion
Trial Locations
- Locations (3)
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
Royal Melbourne Hospital🇦🇺Melbourne, Parkville VIC, Australia