Avacostar - A Post Authorization Safety Study (PASS) to Evaluate the Incidence of Safety Events of Interest in Patients Treated With Avacopan for ANCA-associated Vasculitis (AAV)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 507
- 试验地点
- 59
- 主要终点
- To evaluate the incidence of defined MESIs
研究概览
简要总结
The Avacostar PASS is a non-interventional, multi-national, prospective cohort study that will collect data from 2 cohorts of patients: those treated with avacopan for active severe AAV, and a second cohort treated with a cyclophosphamide or rituximab-based induction regimen without avacopan for active severe AAV. The overall study duration is anticipated to be up to 7 years, including a recruitment period of approximately 3 years.
详细描述
The Avacostar PASS is a non-interventional, multi-national, prospective cohort study that will collect data from 2 cohorts of patients: those treated with avacopan for active severe AAV, and a second cohort treated with a cyclophosphamide or rituximab-based induction regimen without avacopan for active severe AAV. The overall study duration is anticipated to be up to 7 years, including a recruitment period of approximately 3 years. Enrolled patients will be followed until the last patient last visit (LPLV) milestone, which will be 4 years after the last participant is enrolled.
Germany and the United Kingdom (UK) have been selected for the study. Additional countries may be considered according to availability of avacopan and suitability for the study. Patients will be enrolled prospectively, but up to 6 months of data may be collected retrospectively if necessary. Baseline visit is defined as the day that induction treatment (avacopan or non-avacopan standard of care (SoC) cyclophosphamide or rituximab) is started for active severe AAV. Patients who started avacopan/SoC induction therapy for active severe AAV within 6 months of the enrolment visit and fulfil eligibility criteria may be enrolled in the PASS. Individual participant follow-up data will be collected periodically at routine clinic visits until the LPLV, which will be 4 years after the last participant is enrolled.
The primary objective of the study is:
To evaluate the incidence of defined Medical Events of Special Interest (MESIs) in patients with AAV commencing avacopan.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of AAV (MPA or GPA), as determined by the Investigator according to their usual practice.
- •Active, severe AAV at the time of commencing avacopan or non-avacopan SoC induction therapy, in the opinion of the Investigator.
- •Age ≥18 years of either sex.
- •Has provided written informed consent.
- •Has commenced within the previous 6 months, or is planned to commence avacopan, cyclophosphamide or rituximab for the treatment of severe, active AAV outside of an interventional clinical study.
排除标准
- •Concurrent participation in an interventional study, unless prospectively discussed and agreed with the Medical Monitor.
结局指标
主要结局
To evaluate the incidence of defined MESIs
时间窗: up to 7 years
To evaluate the incidence of defined MESIs in patients with AAV commencing avacopan. MESIs include liver injury, cardiac safety, serious infection, and malignancy.
次要结局
- Incidence rates of AE in the avacopan and non-avacopan groups(up to 7 years)
- AEs leading to discontinuation of therapy in the avacopan group(up to 7 years)
- Change in AST over time in the avacopan and non-avacopan groups(up to 7 years)
- Change in WBC over time in the avacopan and non-avacopan groups(up to 7 years)
- Change Albumin over time in the avacopan and non-avacopan groups(up to 7 years)
- Incidence rates of MESIs in the avacopan group compared to the non-avacopan group,(up to 7 years)
- Use of concomitant immunosuppression over time and cumulative by treatment group,(up to 7 years)
- Duration of GC-free periods(up to 7 years)
- Proportion of GC-free patients over time by treatment group.(up to 7 years)
- Incidence of ADRs in the avacopan group(up to 7 years)
- Change in eGFR over time in the avacopan and non-avacopan groups(up to 7 years)
- Change in ALT over time in the avacopan and non-avacopan groups(up to 7 years)
- Change in billirubin over time in the avacopan and non-avacopan groups(up to 7 years)
- Incidence of SADRs in the avacopan group(up to 7 years)
- Incidence of MESIs in the non-avacopan group(up to 7 years)
- Incidence rates of SAEs and MESIs in the avacopan group compared to the non-avacopan group,(up to 7 years)
- Duration of treatment with avacopan by reason for treatment discontinuation.(up to 7 years)
- Incidence of SAEs in the avacopan and non-avacopan groups(up to 7 years)
- Change in IgG over time in the avacopan and non-avacopan groups(up to 7 years)
- Change in CPK over time in the avacopan and non-avacopan groups(up to 7 years)
- Change in VDI scores over time in the avacopan and non-avacopan groups(up to 7 years)
- Time to first flare over time in the avacopan and non-avacopan groups(up to 7 years)