An Open-Label, Safety, Tolerability, and Proof-of-Concept Study of Oral BCX9930 Therapy in Subjects With Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, or Primary Membranous Nephropathy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 3
- 主要终点
- Percent Change From Baseline in 24-hour uPCR at Week 12
研究概览
简要总结
The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non 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
- •Body weight ≥ 40 kilograms (kg)
- •Primary diagnosis of C3G, IgAN, or PMN confirmed by central pathology review
- •An estimated glomerular filtration rate (eGFR) ≥ 50 milliter per minute per 1.73 meter square (mL/min/1.73 m^2) (or ≥ 30 mL/min/1.73 m^2 after Data Monitoring Committee [DMC] recommendation)
- •Receiving treatment with a stable, maximum recommended or maximum tolerated dose of an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) for at least 60 days prior to the Day 1 Visit
- •Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series
排除标准
- •Known congenital deficiency of C1s, C1r, C1q, C2, or C4
- •History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation
- •Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition
- •History of malignancy within 5 years prior to the screening visit
- •Active serious bacterial, viral, or fungal infection or any other serious infection within 14 days of screening
- •Treatment with any systemic immunosuppressive or immunomodulatory therapy within 90 days OR anti-CD20 antibody therapies (eg, rituximab) within 180 days prior to the screening visit
- •Treatment with renin inhibitors (eg, aliskiren) or sodium-glucose-cotransporter 2 (SGLT2) inhibitors within 60 days prior to Day 1
研究组 & 干预措施
C3G cohort
Approximately 14 eligible participants with C3G will be enrolled.
干预措施: BCX9930 (Drug)
IgAN cohort
Approximately 14 eligible participants with IgAN will be enrolled.
干预措施: BCX9930 (Drug)
PMN cohort
Approximately 14 eligible participants with PMN will be enrolled.
干预措施: BCX9930 (Drug)
结局指标
主要结局
Percent Change From Baseline in 24-hour uPCR at Week 12
时间窗: Baseline, Week 12
Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.
Percent Change From Baseline in 24-hour uPCR at Week 24
时间窗: Baseline, Week 24
Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.
次要结局
- Change From Baseline in Estimated Glomerular Filtration Rate(Baseline, Weeks 12, 24)
- Number of Participants With a Treatment-emergent Adverse Event (TEAE)(From first dose up to safety follow-up period (Week 28))
- Percent Change From Baseline in 24-hour Urinary Protein Excretion(Baseline, Weeks 12, 24,)
- Number of Participants Who Discontinued Due to a TEAE(From first dose up to safety follow-up period (Week 28))
- Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality(From first dose up to safety follow-up period (Week 28))
- Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)(From first dose up to safety follow-up period (Week 28))
- Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE(From first dose up to safety follow-up period (Week 28))