Alkali Therapy in Subjects With Sickle Cell Disease (SCD) - Evaluation of Efficacy, Safety, and Beneficial Effects
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- Serum bicarbonate level
研究概览
简要总结
The objective of this study is to assess the effect of alkali administration on bicarbonate and potassium levels in patients with Sickle Cell Disease (SCD) and depressed serum bicarbonate levels. The study is a prospective non-blinded evaluation of tolerability and efficacy of alkali repletion with 4 weeks of observation and two sequential 4 week courses of escalating oral sodium bicarbonate treatment.
详细描述
Primary Objective:
To assess the effect of alkali administration on bicarbonate and potassium levels in patients with SCD and depressed serum bicarbonate levels.
Secondary Objectives:
To assess the effect of alkali administration on improvement in hemolysis and on sequelae of impaired kidney function, ie, LDH, Hgb, reticulocyte count, red cell half-life, and muscle strength, Vitamin D levels, and markers of bone turnover, respectively.
To assess the influence of alkali administrations on markers of kidney tubule inflammation.
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
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Sickle cell disease patients with HbSS
- •eGFR <90 ml/min/1.73m2 (determined by abbreviated 4 variable modification MDRD equation) and/or measured urinary albumin to creatinine ratio >30mg/g.
- •Age ≥18 years
排除标准
- •Previous chronic treatment with alkali (including sodium bicarbonate, calcium carbonate or baking soda)
- •Bicarbonate level >25 mEq/L
- •Decompensated heart failure
- •Uncontrolled systolic blood pressure >140 mm/Hg (the cutoff for systolic hypertension in SCD is lower than in non-SCD)
- •Moderate-to-severe lower extremity edema
- •Projected progression to ESRD within 6 months
- •Kidney transplantation
- •Treatment with immunosuppressives within the last 3 months
- •Vasoocclusive (VOC) within 1 week of study entry
- •Active (open) leg ulcer
- •Change in hydroxyurea dose within the last 3 months, unless a self-limited interruption of a stable dose
- •Blood transfusion within 8 weeks, unless on chronic transfusions
- •Pregnancy
- •Inability to give informed consent
研究组 & 干预措施
Sodium Bicarbonate
Patients will be monitored at baseline bi-weekly intervals for 12 weeks, the first 4 weeks to establish a stable baseline, followed by 8 weeks of alkali therapy, as follows:
干预措施: Sodium Bicarbonate (Drug)
结局指标
主要结局
Serum bicarbonate level
时间窗: 12 weeks
To assess the effect of alkali administration on bicarbonate and potassium levels in patients with SCD and depressed serum bicarbonate levels.
次要结局
- Hemolysis markers(12 weeks)
研究者
Jane Little
Director, Sickle Cell Disease Program
University Hospitals Cleveland Medical Center