2023-508754-26-00已完成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
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Safety will be evaluated by the incidence of AEs, serious adverse events (SAEs), laboratory test results (urinalysis, chemistry, hematology), changes in vital signs, physical examination, and echocardiogram (ECHO), X-rays/QCT results, and clinical hip assessment.
研究概览
简要总结
Primary Objective: To evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Must have completed 24 months (± 14 days) of BMN 111 treatment in Study 111-
- •Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any researchrelated procedure. Also, subjects under the age of 18 are willing and able to provide written assent (if required by local regulations or the IRB/EC) after the nature of the study has been explained and prior to performance of any researchrelated procedure.
- •If sexually active, is willing to use a highly effective method of contraception while participating in the study
- •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
- •Are willing and able to perform all study procedures as physically possible
- •Caregivers are willing to administer daily injections to the subjects and complete the required training – not applicable in France
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 the Baseline visit or planning to become pregnant (self or partner) at any time during the study
- •Current chronic therapy with any of the following restricted medications: • Antihypertensive medications • Angiotensin-converting enzyme (ACE) inhibitors • Angiotensin II receptor blockers • Diuretics • Beta-blockers • Calcium-channel blockers • Cardiac glycosides • Systemic anticholinergic agents • Any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function
结局指标
主要结局
Safety will be evaluated by the incidence of AEs, serious adverse events (SAEs), laboratory test results (urinalysis, chemistry, hematology), changes in vital signs, physical examination, and echocardiogram (ECHO), X-rays/QCT results, and clinical hip assessment.
Safety will be evaluated by the incidence of AEs, serious adverse events (SAEs), laboratory test results (urinalysis, chemistry, hematology), changes in vital signs, physical examination, and echocardiogram (ECHO), X-rays/QCT results, and clinical hip assessment.
次要结局
- Efficacy will be assessed by change from baseline in height growth velocity (annualized to cm/year), height, final adult height, growth parameters, and in body proportions. These changes will be assessed by anthropometric measurements and measurement ratios. Growth parameters (anthropometric measurements) may include but are not limited to height, standing height, sitting height, weight, head circumference, upper and lower arm and leg length, and arm span.
- Body proportion measurements may include but are not limited to upper: lower body segment ratio, upper arm: forearm length ratio, upper leg: lower leg length ratio, and arm span: standing height ratio. Final adult height defined as the height achieved at 16 years of age for females, and 18 years of age for males.
研究者
BioMarin Pharmaceutical Inc.
Scientific
Biomarin Pharmaceutical Inc.
研究点 (1)
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