Last updated on February 2020

Phase 1/2 Lentiviral Vector Gene Therapy - The GuardOne Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease


Brief description of study

This is an adaptive, multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are 16 and 35 years of age and postpubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in plasma and PBLs).

Detailed Study Description

The duration of each subject's participation in this study will be approximately 68 weeks (or 1 year, 16 weeks), comprised of a five study periods (Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up). During the Screening Period (up to 60 days), written informed consent (and assent, if applicable) will be obtained and the subject will complete other Screening procedures to confirm study eligibility. Once study eligibility is confirmed, subjects will enter the Baseline Period (up to 3 days) during which time assessments will be performed to establish a pre-transplant baseline. Once baseline assessments are complete, the subject will enter the Pre-transplant Period (approximately 6-8 weeks) during which time mobilization, apheresis, AVR-RD-02 drug product preparation and testing for release, and planned conditioning regimen administration will take place. Enzyme replacement therapy must be discontinued at least 2 weeks before the scheduled transplant day. Following completion of the Pre-transplant Period, the subject will enter the Transplant Period (1 day) during which AVR-RD-02 infusion will take place. After AVR-RD-02 infusion, the subject will enter the Post-transplant Follow-up Period (approximately 52 weeks), during which time periodic safety and efficacy assessments will be performed to assess measures of safety, engraftment, and clinical response post-transplant. Post-transplant follow-up will occur at the following time points: Week 1 (Days 1 through 7), Week 2 (Days 10 and 14), Week 4 (Day 28), Week 8 (Day 56), Week 13 (Day 91), Week 26 (Day 182), Week 39 (Day 273), and Week 52 (Day 364). During the post-transplant period, subjects will not receive ERT unless pre-specified clinical criteria, which suggest the need for ERT initiation, are met.

After study completion, subjects enrolled will continue periodic safety and efficacy assessments for approximately 14 years (for a total of 15 years post-transplant follow-up) by way of a follow-up study to AVRO-RD-02-201.

Clinical Study Identifier: NCT04145037

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Royal Melbourne Hospital

Melbourne, Australia
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