Pilot Study of ALLN-177 in Adult and Pediatric Subjects Aged 12 Years or Older With Enteric or Primary Hyperoxaluria and Hyperoxalemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 8
- 主要终点
- Change in plasma oxalate
研究概览
简要总结
Evaluate the efficacy and safety of ALLN-177 in reducing plasma and urinary oxalate levels in adult and pediatric patients with enteric hyperoxaluria and hyperoxalemia or primary hyperoxaluria
详细描述
Phase 2, multi-center, open label, single arm study to evaluate the effect and safety of ALLN-177 in adult and pediatric patients aged 12 and older with enteric hyperoxaluria and hyperoxalemia or primary hyperoxaluria .
Approximately 15-20 evaluable subjects are planned to be enrolled in the study. Eligible subjects will administer ALLN-177 with each meal/snack 5 times per day for 12 consecutive weeks.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed a informed consent form or an assent
- •Aged 12 or older with body weight ≥ 35kg
- •History of primary hyperoxaluria or enteric hyperoxaluria associated with a known underlying enteric disorder associated with malabsorption (e.g., bariatric surgery, Crohn's disease, short bowel syndrome, or other malabsorption syndrome)
- •Urinary oxalate ≥ 40mg/24h (normalized for body surface area in children) at Screening in patients with eGFR >15 mL/min/1.73m2
- •In patients with enteric hyperoxaluria, eGFR < 45mL/min/1.73m2 at Screening
- •In patients with enteric hyperoxaluria, plasma oxalate > 5µmol/L at Screening
- •Patients on dialysis, must be stable for greater than 3 months
排除标准
- •Unable or unwilling to discontinue Vitamin C supplementation
研究组 & 干预措施
ALLN-177
ALLN-177 7,500 units (2 capsules)
干预措施: ALLN-177 (Drug)
结局指标
主要结局
Change in plasma oxalate
时间窗: on 12 weeks of treatment
Efficacy will be assessed based on change from baseline in plasma oxalate
次要结局
- Change in 24-hr urinary oxalate excretion(on 12 weeks of treatment)