2024-511544-36-00招募中2 期
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
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 7
- 试验地点
- 3
- 主要终点
- The primary efficacy endpoint is the proportion of subjects with reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan.
研究概览
简要总结
To evaluate the efficacy of pegcetacoplan in improving the underlying pathophysiology of complement 3 glomerulopathy (C3G)/immune complex membranoproliferative lomerulonephritis (IC-MPGN) after 12 weeks of treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 主要目的
- Part B long-term extension
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •At least 18 years of age at screening
- •Willing and able to provide written informed consent
- •Able to understand and willing to comply with all scheduled procedures and other requirements of the study in the opinion of the investigator
- •Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration
- •Must have clinical and pathologic evidence of recurrent C3G or ICMPGN, as evidenced by all of the following: a. A diagnosis of C3G or IC-MPGN, with at least 2+ staining for C3c in the renal allograft, confirmed by a central pathologist, based on the screening renal allograft biopsy b. C3G or IC-MPGN must be primary and not secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, autoimmunity, chronic antibody-mediated rejection, chronic thrombotic microangiopathy, or a medication)
- •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 nonresponders to vaccination.
- •Women of childbearing potential, defined as any women who have experienced menarche and who are not permanently sterile or postmenopausal, must each have a negative blood pregnancy test at screening (and negative urine pregnancy at Visit 4) and must agree to use protocol-defined methods of contraception from screening through 12 weeks after receiving last dose of pegcetacoplan
- •Men must agree to use protocol-defined methods of contraception and agree to refrain from donating semen from screening through 12 weeks after receiving last dose of pegcetacoplan
排除标准
- •Absolute neutrophil count <1000 cells/mm3 during screening (not including Day 1)
- •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
- •Women who are pregnant, or who are currently breastfeeding
- •Inability to cooperate or any condition that, in the opinion of the investigator, could increase the subject’s risk by participating in the study or confound the outcome of the study
- •Evidence of drug or alcohol abuse or dependence, in the opinion of the investigator
- •Known or suspected hereditary fructose intolerance.
- •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: a. Cured basal or squamous cell skin cancer b. Curatively treated in situ disease c. 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
结局指标
主要结局
The primary efficacy endpoint is the proportion of subjects with reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan.
The primary efficacy endpoint is the proportion of subjects with reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan.
次要结局
- The proportion of subjects with reduction in C3c staining on renal biopsy after 52 weeks of treatment
- The proportion of subjects with stabilization or improvement in estimated glomerular filtration rate (eGFR), over time
- The proportion of subjects with stabilization or improvement of serum creatinine concentration over time
- Changes from baseline biopsy in C3c staining over time
- Changes and percentage changes from baseline in eGFR and serum creatinine concentration over time
- Safety: The number and incidence of treatment-emergent adverse events (TEAEs)
- Safety: The change from baseline in vital signs measurements and clinical laboratory test and ECG results
研究者
Clin. Development and Med. Affairs
Scientific
Apellis Pharmaceuticals Inc.
研究点 (3)
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