NCT04387448终止2 期
A Phase 2a Multiple Ascending, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GFB-887, a TRPC5 Channel Inhibitor, in Patients With Diabetic Nephropathy, Focal Segmental Glomerulosclerosis, and Treatment-Resistant Minimal Change Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 96
- 试验地点
- 72
- 主要终点
- Percentage change in Urine Albumin-to-Creatinine Ratio (UACR)
研究概览
简要总结
This is a phase 2a study evaluating the safety and tolerability of multiple ascending doses of GFB-887 in patients with diabetic nephropathy (DN), focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD).
详细描述
Approximately 125 patients will be enrolled in this study across the United States. Patients with DN and FSGS/TR-MCD will be randomized in 3 ascending dose cohorts to receive either GFB-887 or placebo.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients:
- •Male or female 18-75 years of age, of any race, at the time of signing informed consent.
- •Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2 at Screening.
- •Currently receiving an angiotensin-converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB).
- •For DN patients:
- •Diagnosis of type 2 diabetes with glycated hemoglobin (HbA1c) level ≤11% at Screening.
- •UACR ≥ 150 mg/g.
- •For FSGS/TR-MCD patients:
- •Diagnosis of FSGS based on either biopsy or genetic testing or TR-MCD based on biopsy.
- •UPCR ≥ 1.0 g/g.
排除标准
- •All patients:
- •Evidence of another (non-DN, non-FSGS/TR-MCD, respectively) kidney disease.
- •History of malignancy, unless in remission for at least 5 years other than adequately treated basal cell or squamous cell skin cancer, cervical carcinoma in situ, or prostate cancer not expected to require treatment over the course of the study.
- •History of any organ or bone marrow transplant, including kidney grafts.
- •History of alcoholism or drug/chemical abuse within 12 months prior to Screening.
- •For DN patients:
- •Renal disease that requires immunosuppressive therapy (currently, or in the past).
- •Body mass index (BMI) >45 kg/m
- •For FSGS/TR-MCD patients:
- •Currently on calcineurin inhibitors or history of resistance to calcineurin inhibitors.
- •Body mass index (BMI) >40 kg/m
- •Known history of severe or chronic hepatobiliary disease.
研究组 & 干预措施
GFB-887 multiple ascending dose (MAD) active
Experimental
GFB-887 active once-daily dosing
干预措施: GFB-887 (Drug)
GFB-887 MAD placebo
Placebo Comparator
GFB-887 placebo once-daily dosing
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage change in Urine Albumin-to-Creatinine Ratio (UACR)
时间窗: 12 weeks
Percentage change in Urine Protein-to-Creatinine Ratio (UPCR)
时间窗: 12 weeks
次要结局
- Proportion of FSGS/TR-MCD patients achieving a modified partial remission(12 weeks)
- Percentage change in 24-hour urine protein excretion(12 weeks)
- Percentage change in 24-hour urine albumin excretion(12 weeks)
- Proportion of patients (DN or FSGS/TR-MCD) with a UACR/UPCR decrease of at least 50% of baseline(12 weeks)
- Proportion of FSGS/TR-MCD patients achieving a complete remission(12 weeks)
- Incidence and severity of adverse events(12 weeks)
- Incidence of clinically significant changes in 12-lead electrocardiogram (ECG) parameters, vital signs measurements, and physical examinations(Approximately 12 weeks)
- Proportion of patients (DN or FSGS/TR-MCD) with a UACR/UPCR decrease of at least 30% of baseline(12 weeks)
- Incidence of clinically significant changes in laboratory parameters(12 weeks)
- Plasma PK parameters: area under the plasma concentration-time curve (AUC)(12 weeks)
- Proportion of patients (DN or FSGS/TR-MCD) with a UACR/UPCR decrease of at least 40% of baseline(12 weeks)
- Plasma pharmacokinetics (PK) parameters: maximum observed plasma concentration (Cmax)(12 weeks)
- Plasma PK parameters: time of the observed plasma concentration (Tmax)(12 weeks)
研究者
研究点 (72)
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