NCT07073014Enrolling By Invitation3 期
A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 140
- 试验地点
- 38
- 主要终点
- Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
研究概览
简要总结
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays
- •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 study-related procedure
- •Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study
- •If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study
- •Participants are willing and able to perform all study procedures as physically possible
- •Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training
排除标准
- •Permanently discontinued study treatment in the studies 111-303 or 111-212
- •Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care
- •Taking or planning to take any prohibited medications
- •Planned or expected to have limb-lengthening surgery during the study period
- •Planned or expected bone-related surgery during the study period
- •Require any investigational agent prior to completion of study period
- •Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
- •Have known hypersensitivity to vosoritide or its excipients
- •Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study
- •Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
研究组 & 干预措施
Vosoritide
Experimental
Open-label active drug
干预措施: Vosoritide (Drug)
结局指标
主要结局
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
时间窗: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
As measured by change from baseline in height Z-score
次要结局
- Evaluate long-term effect of vosoritide treatment on bone age versus chronological age(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- Evaluate long-term effect of vosoritide treatment on bone quality(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- Evaluate the maintenance effect of vosoritide treatment on standing height(Baseline to week 52)
- Evaluate long-term effect of vosoritide treatment on growth velocity(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- Evaluate the maintenance effect of vosoritide treatment on standing height Z-score(Baseline to week 52)
- Evaluate long-term effect of vosoritide treatment on body proportionality(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- Evaluate long-term changes in Health-Related Quality of Life (HRQoL)(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- To monitor the frequency of seizures(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
- To monitor the incidence of otitis media(Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males)
研究者
研究点 (38)
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