A Sequential Phase 2/3, Single-Arm, Open-Label Study in Adults Followed by a Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults and Adolescents with Focal Segmental Glomerulosclerosis
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 97
- 试验地点
- 32
- 主要终点
- Phase 2: Evaluate the efficacy of pegcetacoplan in terms of change from baseline in log-transformed urine protein to creatinine ratio (uPCR) Phase 3: Change from baseline in log-transformed urine protein to creatinine ratio (uPCR)
研究概览
简要总结
Primary objective for Phase 2: To assess the efficacy of twice-weekly SC doses of pegcetacoplan in adult participants with FSGS on the basis of a reduction of proteinuria
Primary objective for Phase 3: To assess the efficacy of twice-weekly SC doses of pegcetacoplan compared with placebo in participants with FSGS on the basis of a reduction of proteinuria
入排标准
- 年龄范围
- 0 years 至 65+ years（65+ Years, 18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Age - Phase 2: adults aged ≥18 years - Phase 3: adults aged ≥18 years; if and where approved, adolescents (aged 12--17 years) at the time of signing the informed consent and assent form
- •Weight ≥30 kg and ≤100 kg at screening
- •FSGS diagnosis - Phase 2: primary, genetic, or undetermined FSGS diagnosed by kidney biopsy - Phase 3: primary, genetic, or undetermined FSGS diagnosed by kidney biopsy or by recognized podocyte genetic mutation"
- •At least 1.5 g/day of proteinuria on a screening 24-hour urine collection and a uPCR of at least 1.5 g/g in at least 2 FMU samples collected during screening"
- •Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m^2
- •Stable regimen for FSGS treatment for at least 12 weeks prior to the first dose of IP with no planned or anticipated adjustments or dose changes to the stable treatment regimen"
排除标准
- •1 Previous exposure to pegcetacoplan
- •2 Evidence of improving kidney disease in the 8 weeks prior to screening or during the screening period according to available data
- •3 FSGS secondary to another condition (eg, infectious, diabetic, drug-induced, obesity, prematurity, sickle-cell, vesicoureteral reflux, congenital anomalies of the kidney, and urinary tract)
- •4 Type 1 or uncontrolled (HbA1C ≥8%) type 2 diabetes mellitus
- •5 History of kidney transplant
- •6 Current or prior diagnosis of HIV, hepatitis B, or hepatitis C infection or positive serology or viral load during screening that is indicative of active infection with any of these viruses
- •7 Hypersensitivity to pegcetacoplan or to any of the excipients
- •8 Significant other kidney disease that would, in the opinion of the investigator, confound interpretation of study results
- •9 Use of rituximab, belimumab, or any approved or investigational anticomplement therapy within 5 half-lives of that product prior to the screening period
研究组 & 干预措施
ASPAVELI 1 080 mg solution for infusion
干预措施: ASPAVELI 1 080 mg solution for infusion (Drug)
Pegcetacoplan matching placebo is a sterile solution of 10 mM acetate buffer, pH 5.0, containing 4.1% sorbitol in a single-use glass vial.
干预措施: Pegcetacoplan matching placebo is a sterile solution of 10 mM acetate buffer, pH 5.0, containing 4.1% sorbitol in a single-use glass vial. (Drug)
结局指标
主要结局
Phase 2: Evaluate the efficacy of pegcetacoplan in terms of change from baseline in log-transformed urine protein to creatinine ratio (uPCR) Phase 3: Change from baseline in log-transformed urine protein to creatinine ratio (uPCR)
Phase 2: Evaluate the efficacy of pegcetacoplan in terms of change from baseline in log-transformed urine protein to creatinine ratio (uPCR) Phase 3: Change from baseline in log-transformed urine protein to creatinine ratio (uPCR)
次要结局
- Phase 2: Evaluate the efficacy of pegcetacoplan in terms of change from baseline in log-transformed urine albumin to creatinine ratio (uACR) Phase 3: Change from Baseline in log-transformed urine protein to creatinine ratio (uPCR); Proportion of participants achieving Complete Remission; Slope of estimated Glomerular Filtration Rate (eGFR)
研究者
Clinical Trial
Scientific
Apellis Pharmaceuticals Inc.