ADRENL - ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children, Pilot Study
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 主要终点
- Remission of proteinuria
研究概览
简要总结
We propose to study the use of purified porcine Acthar Gel (ACTHAR, Mallinckrodt Pharmaceuticals) for treatment of steroid resistance nephrotic syndrome (SRNS) in a prospective pilot study. We plan to enroll 25 children between the ages of 2 to 21 years. Children fulfilling strict inclusion criteria, whose parents agree to written informed consent after institutional IRB approval for the study, will be enrolled. Purified porcine Acthar Gel will be administered SQ to all children using a defined treatment protocol for a period of six months. Renal function, urine protein excretion, serum albumin levels, blood pressure and growth parameters will be monitored closely on all patients. Baseline urine protein excretion will be compared to end of treatment levels to determine successful response to therapy. There will be an 18 month enrollment period, 6 month treatment period and a 12 month follow-up period.
详细描述
Treatment Protocol:
Patients who fulfill inclusion criteria and who agree to participate after signing informed consent and assent forms will be treated with the following protocol:
Acthar Gel will be dosed by body surface area (BSA) using the Dubois Method. The dose of Acthar Gel will be 80 units/1.73 m2 per dose administered subcutaneously (SQ) twice a week on Mondays and Thursdays.
Week 1 to 2:
During week 1, patients will receive 50% of initial calculated dose twice a week.
研究设计
- 研究类型
- Interventional
- 干预模型
- Sequential
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL
- •Calculated GFR (eGFR) using modified Schwartz formula of > 50 mls/min/m
- •(Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL)
- •A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study.
- •Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy
排除标准
- •Patients with an inherited or genetic disorder presenting with nephrotic syndrome (eg: NPHS 1 & 2 defects, WT-1 mutations, α actinin 4 mutation, TRP-6 mutation).
- •Presence of diabetes or severe (stage 2) uncontrolled hypertension.
- •Any metabolic condition that specifically precludes the use of Acthar Gel for treatment.
- •Pregnancy or unwilling to agree to contraception which may include abstinence.
- •eGFR <50 mls/min/m2
研究组 & 干预措施
Acthar Gel treatment group
Participants will be treated with 'Acthar Gel 80 UNT/ML Injectable Solution'. Initial dose for week 1 will be 50% of 80 units, injected twice per week. Week 2 is 75% of 80 units, week 3 and throughout treatment period (6 months in total) will be 80 units/ml twice per week.
干预措施: Acthar Gel 80 UNT/ML Injectable Solution (Drug)
结局指标
主要结局
Remission of proteinuria
时间窗: 18 months
Partial remission: Greater than or equal to 50% reduction in proteinuria when compared to baseline. Complete remission: Greater than or equal to 90% reduction in proteinuria when compared to baseline or Urine Protein Creatinine ratio less than 0.5.
次要结局
- Serum Lipids(18 months)
- Blood pressure(18 months)
- Serum Creatinine(18 months)
- BMI(18 months)
- Serum Glucose(18 months)
- Quality of life measures obtained via survey(18 months)
研究者
Mohammed Khurram Faizan
Principal Investigator
Rhode Island Hospital