A Long-Term Follow-Up Study of Subjects With Fabry Disease Who Previously Received Ex-Vivo, Lentiviral Vector-Mediated Gene-Modified Autologous Cell Therapy AVR-RD-01 in Study AVRO-RD-01-201
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- AVROBIO
- 入组人数
- 5
- 试验地点
- 3
- 主要终点
- Presence of replication competent lentivirus (RCL)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 16 Years 至 50 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study.
排除标准
- •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.
结局指标
主要结局
Presence of replication competent lentivirus (RCL)
时间窗: Baseline to Year 15 post gene therapy infusion
Presence of anti-Alpha-galactosidase A (AGA) antibodies
时间窗: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by vital signs
时间窗: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests
时间窗: Baseline to Year 15 post gene therapy infusion
Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA)
时间窗: Baseline to Year 15 post gene therapy infusion
Incidence of clinically significant AEs and SAEs
时间窗: Baseline to Year 15 post gene therapy infusion
次要结局
- Change from baseline in eGFR(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)
- 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)