Phosphate Binder Therapy and Chronic Kidney Disease in Children
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 160
- 试验地点
- 32
- 主要终点
- iFGF23 levels
研究概览
简要总结
We will conduct a 12-month, double-blind, randomized, placebo-controlled trial to assess the effects of therapy with ferric citrate (FC) on changes in intact FGF23 levels (iFGF23, primary endpoint) in 160 pediatric patients (80 in each of the two arms) aged 6-18 years of either sex with chronic kidney disease (CKD) stages 3-4 and age-appropriate normal serum phosphate levels. Participants will be randomized to one of the two groups: 1) FC or 2) FC placebo. Participants will be recruited from 20 core clinical sites.
详细描述
We will conduct a double-blind, randomized, placebo-controlled trial to assess the effects of therapy with ferric citrate (FC) on changes in intact FGF23 levels (iFGF23, primary endpoint) aged 6-18 years of either sex with chronic kidney disease (CKD) stages 3-4 and age-appropriate normal serum phosphate levels. Participants will be randomized to one of the two groups: 1) FC or 2) FC placebo. Participants will be recruited from 20 core clinical sites.
Schedule of Intervention: During the 12-month trial, participants will be given a daily fixed weight-based dose of FC.
Schedule for data collection/analyses to be performed:
Blood for primary outcome assessments will be collected at screening, baseline and at months 3, 6, 9, 12. Blood for safety assessments will be collected at the the months 1, 2, 3, 6, 9, 12.
The primary analyses for this 2-arm trial will compare log-transformed iFGF23 values over 12 months between the treatment and the placebo arms. The analysis will use a linear mixed-effects model, including stratification factors CKD stage and urine protein to creatinine ratio, with random participant effects accounting for repeated measurements, and a fixed treatment effect, which interacts with a time indicator (Months 3-12 vs. Baseline/Screening).
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages 6 to 18 years (inclusive);
- •Estimated Glomerular Filtration Rate (GFR) of 15-59 ml/min per 1.73 m2 by modified Chronic Kidney disease in Children (CKiD) under 25 (U25) formula;56
- •Serum phosphate <=5.9 mg/dl;
- •Serum ferritin <500 ng/ml and TSAT <50%;
- •For those patients treated with growth hormone, calcitriol, nutritional vitamin D, iron, and/or erythropoiesis-stimulating agents (ESAs) such treatments must have stable dosing for at least 2 weeks prior to screening;
- •Able to swallow tablets;
- •Able to eat at least two meals a day;
- •In the opinion of the investigator, willing and able to follow the study treatment regimen and comply with the site investigator's recommendations.
排除标准
- •Patients currently treated with phosphate binders.
- •History of allergy to all ingredients (including non-medical ingredients) in both products (i.e. investigational product and placebo)
- •Current intestinal malabsorption, documented in the medical record; disease, inflammatory bowel syndrome, and/or Crohn's Disease.
- •Anticipated initiation of dialysis or kidney transplantation within 6 months
- •Current or planned future systemic immunosuppressive therapy
- •Prior solid organ transplantation
- •Receipt of bone marrow transplant within two years of screening
- •Current pregnancy, lactation or female subjects who have reached menarche, unless using highly-effective contraception as outlined in section 7.1.1 of Protocol
- •Patients participating in other interventional study (observational study participation permitted)
- •Poor adherence to medical treatments in the opinion of the investigator
- •Fanconi syndrome
- •Hemochromatosis or laboratory tests indicating possible hemochromatosis or other iron overload (primary or secondary) syndrome
研究组 & 干预措施
Treatment Arm
During the 12-month trial, participants will be given a fixed weight-based dose of Ferric Citrate (FC). The full medication dose will be 3g/day for participants weighing <31 kg, 5g/day for those weighing >31 - <51 kg, and 6g/day for participants >51 kg. These doses will be divided into three doses to be taken with meals.
干预措施: Ferric Citrate (Drug)
Control Arm
During the 12-month trial, participants will be given a fixed weight-based dose of Placebo. The full medication dose will be 3g/day for participants weighing <31 kg, 5g/day for those weighing >31 - <51 kg, and 6g/day for participants >51 kg. These doses will be divided into three doses to be taken with meals.
干预措施: Placebo (Drug)
结局指标
主要结局
iFGF23 levels
时间窗: 12 months
Compared to placebo, active treatment with FC will lower iFGF23 levels
Safety of Ferric Citrate
时间窗: 12 months
Comparing proportion of subjects with AE and SAE between arms
Tolerability of Ferric Citrate
时间窗: 12 months
Compared with placebo, active treatment will be tolerable
次要结局
- Effects on Transferrin Saturation (TSAT)(12 months)
- Effects on PTH and 1,25 D(12 months)
研究者
Isidro Salusky, MD
Distinguished Professor of Pediatrics at the David Geffen School of Medicine at UCLA, Chief of Pediatric Nephrology and Director of the Pediatric Dialysis Program
University of California, Los Angeles