NCT06321601进行中（未招募）3 期
A Phase 3, Open-label, Uncontrolled Single-arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Avacopan in Combination With a Rituximab or a Cyclophosphamide-containing Regimen in Children From 6 Years to < 18 Years of Age With Active ANCA-associated Vasculitis (AAV)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- Amgen
- 入组人数
- 19
- 试验地点
- 47
- 主要终点
- Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)
研究概览
简要总结
The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female children and adolescents from 6 to < 18 years old of age.
- •Clinical diagnosis of Granulomatosis with Polyangiitis (GPA) or microscopic polyangiitis (MPA), consistent with Chapel-Hill Consensus Conference definitions (Jennette et al, 2013).
- •Positive anti-PR3(Anti-Proteinase 3) or anti-MPO(Anti-Myeloperoxidase) antibody documented at Screening or historically. Historical positivity is acceptable (even if Screening is negative) if supported by verifiable lab source documentation obtained during AAV diagnosis or disease course; use the most recent positive result.
- •At least 1 PVAS major item, at least 3 PVAS nonmajor items, or atleast the 2 renal items of proteinuria and hematuria.
- •Estimated glomerular filtration rate (eGFR) of ≥ 15 mL/minute/1.73 m^2 at screening and day
- •Participants must have a bodyweight of ≥ 15 kg at day 1.
排除标准
- •Any other known multisystem autoimmune disease including and not limited to eosinophilic granulomatosis with polyangiitis (EGPA, previously, Churg-Strauss disease), systemic lupus erythematosus, IgA vasculitis / Henoch-Schönlein, Purpura, rheumatoid vasculitis, Sjögren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis.
- •Renal replacement therapy / plasmapheresis: subjects will be excluded who received, require, or initiate CRRT (continuous renal replacement therapy), hemodialysis, any renal dialysis, or plasmapheresis within 14 days prior to Screening or between Screening and Day
- •History of kidney transplantation or is anticipated to require renal transplantation during the study.
- •Alveolar hemorrhage requiring invasive pulmonary ventilation support anticipated to last beyond the screening period of the study.
- •Any medical condition requiring, or expected to require, ongoing treatment with immunosuppressive, therapy (including systemic glucocorticoids) for a non-AAV indication that in the judgment of the investigator, could confound study assessments or interpretation of study results.
- •Female subjects of childbearing potential must have a negative highly sensitive serum pregnancy test at Screening and a negative sensitive urine pregnancy test, on day 1, with results confirmed prior to the first administration of investigational product.
- •Known hypersensitivity or contraindication to avacopan, its excipients, or to any investigational product or required concomitant medication used in this study.
- •Subject likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures (eg, Clinical Outcome Assessments) to the best of the subject and investigator's knowledge.
- •History or evidence of any other clinically significant disorder, condition, or disease (other than those specified above) that, in the investigator's judgment, would pose an unacceptable risk to subject safety, or interfere with study assessments or completion. The investigator may consult the Amgen medical monitor as needed. The rationale for exclusion and any consultation must be documented in the subject's source record.
研究组 & 干预措施
Avacopan
Experimental
Participants will receive avacopan twice-daily (BID) administered as oral tablets or liquid formula for 52 weeks.
干预措施: Avacopan (Drug)
结局指标
主要结局
Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)
时间窗: Week 26
Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS
时间窗: Week 52
次要结局
- Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)(Baseline up to Week 52)
- Taste and Acceptability Score of Avacopan per TASTY Faces Scale(Day 1 and Week 2)
- Proportion of Participants Achieving Disease Remission at Week 26 According to the Birmingham Vasculitis Activity Score (BVAS)(Week 26)
- Proportion of Participants With BVAS of 0 Over Time Through Week 52(Up to Week 52)
- Plasma Concentrations of Avacopan(Day 1 up to Week 52)
- Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)(Day 1 up to approximately Week 60)
- Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS(Week 52)
- Proportion of Participants With PVAS of 0 Over Time Through Week 52(Up to Week 52)
- Number of Glucocorticoid Dosages Administered(Screening up to Week 52)
- Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)(Baseline up to Week 52)
- Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)(Baseline up to Week 52)
- Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity(Baseline up to Week 52)
- Proportion of Participants Across the Taste Score Categories of the TASTY Faces Scale(Day 1 and Week 2)
研究者
研究点 (47)
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