An Open-label, Randomised, Active-controlled, Parallel Group, Multicentre, Phase 3 Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients With Hyperphosphataemia
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 85
- 试验地点
- 38
- 主要终点
- Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 Group
研究概览
简要总结
This is a Phase 3, Open-label, Randomised, Active-controlled, Parallel Group, Multicentre Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients with Hyperphosphataemia. The aim of this Phase 3 clinical study is to demonstrate similar efficacy of PA21 (Velphoro) in paediatric and adolescent patients with CKD, and to provide safety and dosing information for this patient population. The Phoslyra (comparator) group provides information for a descriptive comparison of PA21 against a commonly used calcium-based phosphate binder (calcium acetate).
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
- None
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects 0 to <18 years at time of consent.
- •Subjects with hyperphosphataemia
- •Subjects ≥1 year with CKD Stages 4-5 defined by a glomerular filtration rate <30 mL/min/1.73 m2 or with CKD Stage 5D receiving adequate maintenance haemodialysis (HD) or peritoneal dialysis (PD) for at least 2 months prior to screening.
- •Subjects <1 year must have CKD.
- •Appropriate written informed consent and, where appropriate/required assent, have been provided.
排除标准
- •Subjects with hypercalcaemia at screening
- •Subjects with intact parathyroid hormone (iPTH) levels >700 pg/mL at screening.
- •Subjects who are PB naïve who weigh <5 kg at screening. Subjects receiving stable doses of PBs who weigh <6 kg at screening
- •Subjects requiring feeding tube sizes ≤6 FR (French catheter scale).
- •Subjects with history of major gastrointestinal surgery or significant gastrointestinal disorders.
- •Subjects with hypocalcaemia (serum total corrected calcium <1.9 mmol/L; <7.6 mg/dL) at screening.
- •Subject is pregnant (e.g., positive human chorionic gonadotropin test) or breast feeding.
- •Subject has a significant medical condition(s)
研究组 & 干预措施
PA21 (Velphoro®)
PA21 (Velphoro®), chewable tablets 500 mg iron
PA21 (Velphoro®), chewable tablets 250 mg iron
PA21 (Velphoro®), powder for oral suspension 500 mg iron
PA21 (Velphoro®), powder for oral suspension 250 mg iron
PA21 (Velphoro®), powder for oral suspension 125 mg iron
干预措施: PA21 (Velphoro®) (Drug)
Calcium Acetate (Phoslyra®)
Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.
干预措施: Calcium Acetate (Phoslyra®) (Drug)
结局指标
主要结局
Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 Group
时间窗: From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
Number and Percentage of Participants With Any Treatment Emergent Adverse Event
时间窗: through study completion, up to 34 weeks after treatment start date
Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAEs until end of stage 2. Please refer to the detailed tables included on the Adverse Event Module for specifics.
Number and Percentage of Participants Who Withdrew Due to Treatment Emergent Adverse Events
时间窗: through study completion, up to 34 weeks after treatment start date
Any adverse event Leading to Study Drug Withdrawal is considered.
次要结局
- Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the Phoslyra Group(From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date))
- Participants With Serum Phosphorus Levels Within the Age Related Normal Range in Each Stage(through study completion, up to 34 weeks after treatment start date)
- 25-Hydroxy Vitamin D Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Serum Phosphorus Values at Each Visit(through study completion, up to 34 weeks after treatment start date)
- Unsaturated Iron Binding Capacity Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Change in Serum Phosphorus Level From Baseline to the End of Stage 2 in Both Groups(From baseline to study completion, up to 34 weeks after treatment start date)
- Serum iPTH Levels at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Ferritin Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Fibroblast Growth Factor 23 Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Osteocalcin-CL Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Tartrate-resistant Acid Phosphatase 5b Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Participants With Serum Phosphorus Levels Within the Age-dependent Target Range in Each Stage(through study completion, up to 34 weeks after treatment start date)
- Serum Total Corrected Calcium at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Participants With Sustained Hypercalcaemia(through study completion, up to 34 weeks after treatment start date)
- Transferrin Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Type I Collagen C-Telopeptides Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Serum Total Corrected Calcium-Phosphorus Product at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Iron Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)
- Bone Specific Alkaline Phosphatase Values at Each Time Point and Change From Baseline(From baseline through study completion, up to 34 weeks after treatment start date)