A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

Apellis Pharmaceuticals, Inc.123 个研究点分布在 7 个国家目标入组 124 人2021年11月12日

适应症C3G IC-MPGN C3 Glomerulopathy C3 Glomerulonephritis Complement 3 Glomerulopathy Complement 3 Glomerulopathy (C3G)Complement 3 Glomerulonephritis Dense Deposit Disease DDD Membranoproliferative Glomerulonephritis Membranoproliferative Glomerulonephritis (MPGN)Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

干预措施Pegcetacoplan Placebo

概览

阶段: 3 期
干预措施: Pegcetacoplan
疾病 / 适应症: C3G
发起方: Apellis Pharmaceuticals, Inc.
入组人数: 124
试验地点: 123
主要终点: Randomized Controlled Period: Change From Baseline in Log-Transformed Urine Protein-to-Creatinine Ratio (uPCR) at Week 26
状态: 已完成
最后更新: 3个月前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验相关资讯

概览

简要总结

This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN) on the basis of a reduction in proteinuria.

注册库

clinicaltrials.gov

开始日期

2021年11月12日

结束日期

2025年1月14日

最后更新

3个月前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

Apellis Pharmaceuticals, Inc.

责任方

Sponsor

入排标准

入选标准

•Aged at least 18 years; where approved, adolescents (aged 12-17 years) weighing at least 30 kg may also be enrolled.
•A diagnosis of primary C3G or IC-MPGN (with or without previous renal transplant).
•Evidence of active renal disease, based on one or more of the following:
•In adults or adolescents with a baseline renal biopsy (either one collected during screening or a historic biopsy collected within 28 weeks prior to randomization), at least 2+ C3c staining on the baseline renal biopsy.
•In adolescents not providing a baseline renal biopsy, at least one of the following:
•Plasma sC5b-9 level above the upper limit of normal during screening
•Serum C3 below the LLN during screening
•Presence of an active urine sediment during screening, as evidenced by hematuria with at least 5 red blood cells (RBCs) per high-power field (HPF) and/or red blood cell casts on local or central microscopic analysis of urine.
•Presence of C3 nephritic factor within 6 months of screening, based on central laboratory results or medical history.
•No more than 50% global glomerulosclerosis or interstitial fibrosis on the baseline biopsy for adult participants or adolescent participants providing a baseline biopsy.

排除标准

•Previous exposure to pegcetacoplan.
•C3G/IC-MPGN secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, a systemic autoimmune disease such as systemic lupus erythematosus, chronic antibody-mediated rejection, or a medication), in the opinion of the investigator.
•Current or prior diagnosis of human immunodeficiency virus (HIV), hepatitis B (HBV), or hepatitis C (HCV) infection or positive serology during screening that is indicative of infection with any of these viruses.
•Body weight greater than 100 kg at screening.
•Hypersensitivity to pegcetacoplan or to 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

研究组 & 干预措施

Group 1: Pegcetacoplan administration

Subcutaneous infusion of 20mL (1080 mg), twice weekly (for adults or adolescents \>50kg), and the three other weight-based doses either of 10mL (540mg), 12mL (648mg), or 15mL (810mg)

干预措施: Pegcetacoplan

Group 2: Placebo administration

Subcutaneous infusion of either 10mL, 12mL, 15mL, or 20mL, twice weekly

干预措施: Placebo

结局指标

主要结局

Randomized Controlled Period: Change From Baseline in Log-Transformed Urine Protein-to-Creatinine Ratio (uPCR) at Week 26

时间窗: Baseline (Day -70 to Day 1) to Week 26

Baseline uPCR value was calculated as the average of the uPCR measurements from at least 6 of the 9 first-morning spot urine (FMU) samples collected between the start of screening and Day 1, inclusive. The uPCR values used to calculate baseline included those from the samples collected on Day -2, Day -1, and before dosing on Day 1. In situations where less than 6 samples or more than 9 samples were collected, the average of all collected samples was used for baseline derivation. The difference between treatment groups using a composite contrast of equal-weighted average over Weeks 24, 25, and 26 was estimated.

次要结局

Randomized Controlled Period: Percentage of Subjects Who Achieved the Composite Renal Endpoint at Week 26(Week 26)
Randomized Controlled Period: Change From Baseline in the C3 Glomerulopathy (C3G) Histologic Index Activity Score at Week 26(Baseline (Day 1) and Week 26)
Randomized Controlled Period: Percentage of Subjects With a Reduction of At Least 50% From Baseline in Urine Protein-to-Creatinine Ratio at Week 26(Baseline (Day -70 to Day 1) and Week 26)
Randomized Controlled Period: Percentage of Subjects Who Showed Decrease in C3c Staining on Renal Biopsy From Baseline at Week 26(Baseline (Day 1) and Week 26)
Randomized Controlled Period: Change From Baseline in the Kidney Disease Quality of Life (KDQOL) Score at Week 26(Baseline (Day 1) and Week 26)
Randomized Controlled Period: Change From Baseline in Estimated Glomerular Filtration Rate at Week 26(Baseline (Day 1) and Week 26)
Randomized Controlled Period: Percentage of Subjects Who Achieved Proteinuria <1 Gram (g)/Day at Week 24(Week 24)
Randomized Controlled Period: Percentage of Subjects With Normalization of Serum Albumin Levels at Week 26(Week 26)
Randomized Controlled Period: Percentage of Subjects With Serum C3 Levels Above the Lower Limit of Normal at Week 26(Week 26)
Randomized Controlled Period: Change From Baseline in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score at Week 26(Baseline (Day 1) and Week 26)