Participants enrolled in a study where the individual received AVR-RD-04 will be offered participation in the AVRO-RD-04-LTF01 study. The Baseline visit for the AVRO-RD-04-LTF01 study will likely coincide with the final visit in the parent study. Participants confirmed eligible for the AVRO-RD-04-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for up to 11 years until a total of 15 years have elapsed during which time continued safety, engraftment, and efficacy of AVR-RD-04 treatment will be assessed.
This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-04 treatment in participants who received a single dose administration of lentiviral gene therapy. 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 the initial date of AVR-RD-04 infusion.