临床试验/jRCT2031240290

jRCT2031240290招募中不适用

An observational study of real-world outcomes in Japanese children with achondroplasia initiating Voxzogo before the age of 2 years

BioMarin Pharmaceutical Japan K.K.0 个研究点目标入组 50 人开始时间: 待定最近更新: 2025年5月8日

适应症

试验速览

阶段: 不适用
状态: 招募中
发起方: BioMarin Pharmaceutical Japan K.K.
入组人数: 50
主要终点: -

研究概览研究设计入排标准结局指标研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Observational

入排标准

年龄范围: No limit 至 2age old not（—）
性别: All

入选标准

•Documented diagnosis of ACH as per standard of care.
•Have initiated Voxzogo treatment, or plan to initiate Voxzogo treatment within 3 months, before the age of 2 years, in accordance with local standard clinical practice and local label with full medical record available.
•Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure.
•Have available medical history from the start of Voxzogo treatment, or 6 months of age, whichever is earlier, to enable collection of data elements of interest for the study (including anthropomorphic measurements, medical and surgical outcomes of interest).
•Participants who are on Voxzogo treatment must be enrolled in 111-604 in parallel and provide consent to share safety information via 111-604.

排除标准

•Currently enrolled in an interventional study (e.g., studies which require medical device use or drug therapy or with protocol required procedures) or prior enrollment in another interventional study(s) or receiving investigational agent(s)

结局指标

主要结局

-

Event rate of ACH-related surgeries of interest

次要结局

Event rate of comorbidities
Event rate of CPAP and BiPAP therapy
Changes in anthropometric measurements
Age at achievement of gross motor developmental milestones

研究者

发起方

BioMarin Pharmaceutical Japan K.K.

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