A Multicenter, Comparative Safety and Efficacy Study of ACTHar Gel Alone or in Combination With Oral Tacrolimus to Reduce Urinary Proteinuria in Patients With Idiopathic DNAJB9 Positive Fibrillary Glomerulopathy
试验速览
- 阶段
- 4 期
- 状态
- 进行中（未招募）
- 入组人数
- 34
- 试验地点
- 5
- 主要终点
- The change in UP/CR ratio with treatment of Acthar gel 80 units subcutaneous 2 times a week alone
研究概览
简要总结
A Multicenter, Comparative Safety and Efficacy Study of Acthar gel alone or in combination with oral Tacrolimus to reduce urinary proteinuria in patients with idiopathic DNAJB9 Positive Fibrillary glomerulopathy.
详细描述
A Multicenter, Comparative Safety and Efficacy Study of Acthar gel alone or in combination with oral Tacrolimus to reduce urinary proteinuria in patients with idiopathic DNAJB9 Positive Fibrillary glomerulopathy. This study will be a multi-center, prospective, randomized, open-labeled intervention trial of 34 patients randomized to 52 weeks of ACTHar gel alone or Acthar gel plus oral Tacrolimus.
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
- 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
- •Male/Female age > 18
- •Biopsy proven Fibrillary glomerulonephritis within 3 years of study randomization
- •Stable Maximum Renin-Angiotensin-Aldosterone System inhibition times 4 weeks prior to randomization Note: Maximum RAAS inhibition will be left to the discretion of the site Principal Investigator
- •Estimated Glomerular Filtration Rate > 25 mls/min calculate by the Chronic Kidney Disease-EPI formula
- •Protein/creatinine ratio > 2000 mg/gm 5) Note: If protein/creatinine less than 2000 mg/gm, a formal 24- hour urine collection for total protein can be performed. The total 24-hour protein will need to >/= 2000mg.
- •Blood pressure targeted to < 140/90 at the time of randomization
- •Patients with Monoclonal Gammopathy without history of myeloma will be eligible.
- •Patients with monoclonal staining for fibrillary fibers will be excluded
- •Patients with Type II non-insulin dependent diabetes will be eligible provided the renal biopsy does not show nodular Kimmelstiel Wilson lesions
排除标准
- •Patients with MGUS and history of myeloma will not be eligible
- •Patients with active viral production of either hepatitis B or C as evidence by historical polymerase chain reaction test positive for active viral shedding
- •HIV seropositivity
- •Renal biopsy data with > 50% Interstitial Fibrosis
- •Patient with active or a known history lymphoma
- •Patients with insulin dependent diabetes mellitus will be excluded Note: patients with Type II diabetes mellitus that are well controlled without the need for insulin will be eligible for the study.
- •Patients with Type II non-insulin dependent diabetes will be eligible provided the renal biopsy does not show nodular Kimmelstiel Wilson lesions.
- •Patients receiving steroids, mycophenolate mofetil, cyclophosphamide, Azathioprine or other immunosuppressive agent with 4 weeks of study randomization Note: Washout of these medications will be allowed at the screening visit
- •Patients having received Rituximab or B cell modifying biologic therapy within 6 months of randomization
研究组 & 干预措施
Treatment with Acthar gel
Group 1 - (17 patients) Acthar gel 80 units 2 times a week alone for 12 months of therapy.
干预措施: Acthar Gel 80 UNT/ML Injectable Solution (Drug)
Treatment with combination Acthar gel and Tacrolimus therapy
Group 2 - (17 patients) Acthar get 80 units 2 times a week plus oral Tacrolimus 1.0 mg two times a day titrated to trough Tacrolimus levels between 4-6 ng/ml
干预措施: Acthar Gel 80 UNT/ML Injectable Solution (Drug)
结局指标
主要结局
The change in UP/CR ratio with treatment of Acthar gel 80 units subcutaneous 2 times a week alone
时间窗: 12 months
The change in protein/creatinine ratio in patients with biopsy proven Fibrillary after 12 months of treatment with treated with Acthar gel (80 units subcutaneous 2 times a week) alone
The change in UP/CR ratio with treatment of Acthar gel 80 units subcutaneous 2 times a week in combination with oral Tacrolimus
时间窗: 12 months
The change in protein/creatinine ratio in patients with biopsy proven Fibrillary after 12 months of treatment with treated with Acthar gel 80 units 2 times a week subcutaneous week in combination with oral Tacrolimus 1.0 mg orally two times a day.
次要结局
- The change in Estimated Glomerular Filtration Rate(24 months)
- The relative change in protein/creatinine ratio at 24 months(24 months)
- Percentage of patients complete or partial response(12 months)
- To compare the change in urinary biomarkers of Urinary VEGF 121, 165 189, and206, Urinary MCP-1, Urinary Synaptopodin, Urinary TGF-beta, Urinary Podocalyxin, and Urinary Nephrin(12 months)