A Phase 3, Randomized, Open-label, Parallel-arm, Active-controlled, Multicenter Study to Evaluate Safety and Efficacy of ALXN1850 Versus Asfotase Alfa Administered Subcutaneously in Pediatric Participants (2 to < 12 Years of Age) With Hypophosphatasia (HPP) Previously Treated With Asfotase Alfa
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 43
- 试验地点
- 22
- 主要终点
- Number of Participants with Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.
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
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of HPP documented in the medical records
- •Presence of open growth plates by X-ray during Screening Period
- •Tanner stage 2 or less during the Screening Period
- •Must have been treated with 6 mg/kg/ week of asfotase alfa via SC injection administered as either 2mg/kg 3 times per week or 1 mg/kg 6 times per week for ≥ 6 months before Day
- •Note: participants currently treated with 9 mg/kg (eg, 3 mg/kg 3 times per week) will not be allowed in the study.
排除标准
- •History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, neurological disorders, or any other disorders that are capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data as determined by the Investigator.
- •Diagnosis of primary or secondary hyperparathyroidism
- •Hypoparathyroidism, unless secondary to HPP
- •Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)
- •Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period
- •History of allergy or hypersensitivity to any ingredient contained in asfotase alfa or ALXN1850
- •Body weight < 10 kg during the Screening Period
研究组 & 干预措施
ALXN1850
Starting at Day 1 of the Randomized Evaluation Period participants will receive ALXN1850 for a total of 24 weeks. Participants will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection. During Part A of the OLE Period, participants will have frequent visits over the first 24 weeks; Part B of the OLE Period participants will have visits every 9 months for up to approximately 108 weeks.
干预措施: ALXN1850 (Drug)
ALXN1850
Starting at Day 1 of the Randomized Evaluation Period participants will receive ALXN1850 for a total of 24 weeks. Participants will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection. During Part A of the OLE Period, participants will have frequent visits over the first 24 weeks; Part B of the OLE Period participants will have visits every 9 months for up to approximately 108 weeks.
干预措施: asfotase alfa (Drug)
asfotase alfa
Starting at Day 1 of the Randomized Evaluation Period, participants will receive asfotase alfa for a total of 24 weeks. Participants will receive 6 mg/kg/week of asfotase alfa via SC injection as either 2 mg/kg 3 times per week or 1 mg/kg 6 times per week. Part A of the OLE Period participants will have frequent visits over the first 24 weeks; Part B will have visits every 9 months for up to approximately 108 weeks.
干预措施: asfotase alfa (Drug)
结局指标
主要结局
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
时间窗: Baseline Through Day 169
次要结局
- Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in Bruininks Oseretsky Test of Motor Proficiency, Second Edition (BOT2) Score at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in Peabody Developmental Motor Scales, Third Edition (PDMS-3) Score at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)