Effect of Oral Galactose on the Level of Focal Sclerosis Permeability Factor and Proteinuria in Children With Steroid Resistant Nephrotic Syndrome: A Pilot Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- Focal Segmental Glomerulosclerosis Permeability Factor (FSPF)
研究概览
简要总结
Focal Segmental Glomerulosclerosis (FSGS) is a devastating kidney disease which is difficult to treat and carries a poor prognosis, with 50% of affected children progressing to end stage renal disease (ESRD). The purpose of this study is to investigate oral galactose as a benign treatment for FSGS in children. The investigators hypothesize that galactose, a simple milk sugar thought to bind to the protein factor (FSPF) that causes FSGS thereby inactivating it and stopping it from damaging the kidney, resulting in a reduction in glomerular permeability to albumin and decrease in proteinuria in children with nephrotic syndrome secondary to FSGS.
详细描述
RESEARCH PLAN
A: Primary aims:
- Determine the effect of 4 months of oral galactose administration on the level of FSPF in children with steroid resistant FSGS
- Determine the effect of 4 months of oral galactose administration on the first morning urine protein to creatinine (urine protein: creatinine) ratio and serum albumin level in children with steroid resistant FSGS.
B: Secondary aims:
- Determine the effect of 4 months of oral galactose administration on dose of immunosuppressive medication.
- Assess first morning urine protein: creatinine ratio, serum albumin level, and change in immunosuppression dose at 3 months after discontinuation of oral galactose therapy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 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
- •2-21 years old
- •Biopsy proven FSGS or minimal change with steroid resistance
- •Presence of FSPF (defined as permeability activity >0.5)
- •Presence of nephrotic range proteinuria (urine protein: creatinine ratio >2) at the time of enrollment.
- •Persistent nephrotic range proteinuria despite being on stable immunosuppressive medications (cyclosporine, tacrolimus or mycophenolate mofetil) for at least 12 weeks and/or persistent nephrotic range proteinuria despite being on stable dose of angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) for 12 weeks.
- •Stable serum creatinine (change of less than 0.3 mg/dl) in the prior 3 months.
- •Schwartz estimated (e) glomerular filtration rate (GFR) >60ml/min/1.73m2
排除标准
- •Secondary FSGS
- •Onset of nephrotic syndrome in infancy.
- •Presence of acute renal failure (as defined by acute kidney injury criteria) at the time of enrollment. These children can be enrolled 1 month after resolution of acute renal failure (ARF).
- •Decreasing renal function (persistent increase in serum creatinine of greater than 0.3 mg/dl over baseline in the prior 3 months).
- •Use of another investigational drug
- •Pregnant or unable to comply with contraceptive measures in females of child bearing age
- •eGFR < 60 ml/min per 1.73 m2
- •Children with Galactosemia
- •Children with type 1 or 2 diabetes
研究组 & 干预措施
Galactose
Oral galactose will be given at a dose of 0.2gm/kg/dose twice a day (BID) to a maximum of 15 gm BID for a period of 16 weeks.
干预措施: D-Galactose (Drug)
结局指标
主要结局
Focal Segmental Glomerulosclerosis Permeability Factor (FSPF)
时间窗: 16 weeks
FSPF is reported in relation to its induction of glomerular albumin permeability (Palb) of isolated glomeruli on a range from 0 to 1, with 0 indicative of normal glomeruli and 1 indicative of injury to the permeability barrier. Results will be considered clinically significant if the following criteria is met in response to oral galactose therapy at week 16: Reduction in FSPF to \<0.5 Palb or decrease in FSPF by \> 0.3 Palb.
次要结局
- Number of Participants Achieving Complete or Partial Remission at 16 Weeks(16 weeks)
研究者
Asha Moudgil
Professor of Pediatrics
Children's National Research Institute