NCT02724228进行中（未招募）2 期
A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 9
- 主要终点
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
研究概览
简要总结
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
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
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have completed 24 months of BMN 111 treatment in Study 111-
- •Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority.
- •If sexually active, willing to use a highly effective method of contraception while participating in the study.
- •Females >= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
- •Willing and able to perform all study procedures as physically possible
- •Parents/caregivers willing to administer daily injections to the subjects and complete the required training.
排除标准
- •Requires any investigational agent prior to completion of study period.
- •Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
- •Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason.
- •Permanently discontinued BMN 111 during the 111-202 study.
- •Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study.
- •Current chronic therapy with restricted medications.
研究组 & 干预措施
BMN 111 - Subcutaneous Injection
Experimental
111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen.
干预措施: BMN 111 (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
* Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent serious adverse events
次要结局
- Body Proportions (Efficacy)(Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later)
- Growth Velocity [Efficacy](Until near final adult height is reached)
- Final Adult Height(Up to at least 16 years of age for females and 18 years of age for males)
- Growth Parameters (Efficacy)(Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later)
研究者
研究点 (9)
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