在3-59个月龄的儿童中,沃索里提德治疗:一个跨国,随机,双盲,安慰剂受控,第二阶段试验
Ravi Savarirayan1, William R Wilcox2, Paul Harmatz3
1Murdoch Children's Research Institute, Royal Children's Hospital, and University of Melbourne, Parkville, VIC, Australia.
伏索里提德是一种C型天然尿素类同类物,在5岁以下的儿童中显示出轻度的安全性. 在52周的时间里,治疗导致身高Z得分略有增加.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 骨发育不良症 骨发育不良症
- 药理学 药理学是指药理学的学科.
背景情况:
- 骨生长不良是一种影响骨生长的遗传性疾病.
- 沃索里提德是一种研究用于增长增强的C型天然尿素类型类似物.
- 之前的研究评估了5-18岁儿童的沃索里提德.
研究的目的:
- 为了评估vosoritide的安全性和有效性,在婴儿和5岁以下的患有无质形成症的儿童中使用.
- 为了确定最佳剂量,并评估这个年轻人群的成长结果.
主要方法:
- 一个双盲,随机,安慰剂控制的第二阶段试验.
- 涉及75名年龄在0-59个月的参与者,具有遗传确认的无形质.
- 参与者每天在52周内接受皮下注射伏索里提德或安慰剂.
主要成果:
- 所有参与者都经历了不良事件,大多数是短暂的注射部位反应.
- 报告的严重不良事件发生在7%的沃索里提德组和19%的安慰剂组.
- 高度Z得分变化与基线的平均差异为0.25,有利于vosoritide.
结论:
- 沃索里提德在3-59个月的儿童中表现出轻微的不良事件概况.
- 在52周的治疗后,观察到身高Z得分的适度增长.
- 进一步的研究可能会探索这个年龄组的长期结果.
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