An Open-Label, Randomized, Controlled, Phase 2 Study to Evaluate the Safety and Efficacy of Pegcetacoplan in the Treatment of Post-Transplant Recurrence of C3G or IC-MPGN
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 48
- 主要终点
- Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 12
研究概览
简要总结
This is a Phase 2, multicenter, open-label, randomized, controlled study designed to evaluate the safety and efficacy of pegcetacoplan in patients who have post-transplant recurrence of C3G or IC-MPGN.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
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
- •At least 18 years of age at screening
- •Must have clinical and pathologic evidence of recurrent C3G or IC-MPGN
- •Stable (not improving) or worsening disease, in the opinion of the investigator, in the 2 months preceding the first dose of pegcetacoplan
- •eGFR ≥15 mL/min/1.73 m2, calculated by the Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) creatinine equation for adults
- •No more than 50% glomerulosclerosis or interstitial fibrosis on the screening renal allograft biopsy
- •Stable regimen for recurrent C3G/IC-MPGN for at least 4 weeks prior to the screening renal allograft biopsy and from the time of the screening renal allograft biopsy until randomization
- •Have received required vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae (type B) or agree to receive vaccinations, if applicable vaccination records are not available. Vaccination is mandatory unless documented evidence exists that subjects are non-responders to vaccination.
排除标准
- •Absolute neutrophil count <1000 cells/mm3 during screening
- •Previous treatment with pegcetacoplan
- •Evidence of rejection on the screening renal allograft biopsy that requires treatment
- •Diagnosis or history of HIV, hepatitis B, or hepatitis C infection or positive serology at screening indicative of infection with any of these viruses
- •Weight more than 100 kg at screening
- •Hypersensitivity to pegcetacoplan or any of the excipients
- •History of meningococcal disease
- •Malignancy, except for the following:
- •Cured basal or squamous cell skin cancer
- •Curatively treated in situ disease
- •Malignancy free and off treatment for ≥5 years
- •Significant renal disease in the renal allograft secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, rejection, or a medication) that would, in the opinion of the investigator, confound interpretation of the study results
- •Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives from the last dose of the investigational agent (whichever is longer) prior to screening
- •Known or suspected hereditary fructose intolerance.
研究组 & 干预措施
Group 1
Pegcetacoplan treatment of 1080 mg (sub-cutaneous infusion) twice weekly will be given throughout the entire study.
干预措施: Pegcetacoplan (Drug)
Group 2
No intervention given during the randomized controlled portion of the study (through week 12). After week 12, subjects will receive pegcetacoplan treatment.
干预措施: Pegcetacoplan (Drug)
结局指标
主要结局
Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 12
时间窗: Baseline (Day 1) and Week 12
C3c staining intensity is semi quantitatively graded on a scale of 0 to 3, in which negative, 1+, 2+ and 3+ staining corresponds to scores of 0 to 3, with 0 being absent and 3 being the highest intensity seen on immunofluorescence. Higher scores indicate worse outcome. Reduction in C3c staining was defined as decrease of at least 2 orders of magnitude of intensity from baseline. Baseline was defined as the most recent non-missing measurement prior to first administration of study drug for Group 1 subjects or randomization for Group 2 subjects.
次要结局
- Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 52(Baseline (Day 1) and Week 52)
- Number of Subjects With Shift of C3c Staining From Baseline to Weeks 12 and 52(Baseline (Day 1), Week 12 and Week 52)
- Percentage of Subjects With Stabilization or Improvement in Estimated Glomerular Filtration Rate (eGFR) at Week 52(Baseline (Day 1) and Week 52)
- Percentage of Subjects With Stabilization or Improvement of Serum Creatinine Concentration at Week 52(Baseline (Day 1) and Week 52)
- Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52(Baseline (Day 1) and Week 52)
- Percentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52(Baseline (Day 1) and Week 52)
- Change From Baseline in Serum Creatinine Concentration at Week 52(Baseline (Day 1) and Week 52)
- Percentage Change From Baseline in Serum Creatinine Concentration at Week 52(Baseline (Day 1) and Week 52)