NCT01603095已完成不适用
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 363
- 试验地点
- 20
- 主要终点
- Collection of consistent growth measurements
研究概览
简要总结
Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in future studies sponsored by BioMarin. No study drug is administered.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, willing and able to provide written assent (as needed) after the nature of the study has been explained and prior to performance of any research-related procedure.
- •Aged 0 to <= 17 years, inclusive, at study entry.
- •Have ACH, documented by clinical diagnosis
- •Are ambulatory and able to stand without assistance (not applicable for infants)
- •Are willing and able to perform all study procedures as physically possible.
排除标准
- •Have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia)
- •Have any of the following disorders:
- •Hypothyroidism
- •Insulin-requiring diabetes mellitus
- •Autoimmune inflammatory disease
- •Inflammatory bowel disease
- •Autonomic neuropathy
- •Have an unstable clinical condition likely to lead to intervention during the course of the study, including progressive cervical medullary compression
- •Growth plates have fused
- •Have a history of any of the following:
- •Renal insufficiency
- •Cardiac or vascular disease, including the following:
- •Cardiac dysfunction (abnormal echocardiogram [ECHO] including left ventricle [LV] mass) at Screening Visit
- •Hypertrophic cardiomyopathy
- •Congenital heart disease
- •Cerebrovascular disease, aortic insufficiency
- •Clinically significant atrial or ventricular arrhythmias
- •Current treatment with 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, drugs known to alter renal function that is expected to continue for the duration of the study
- •Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (> 3 months) at any time
- •Have had regular long-term treatment (> 1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
- •Concomitant medication that prolongs the QT/QTc interval within 14 days or 5 half-lives, whichever is longer, before the Screening visit
- •Have used any other investigational product or investigational medical device for the treatment of ACH or short stature
- •Have had bone-related surgery or expected to have bone-related surgery during the study period. Subjects with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae.
- •Have any condition that, in the view of the Investigator, places the patient at high risk of poor compliance with the visit schedule or of not completing the study.
- •Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation
结局指标
主要结局
Collection of consistent growth measurements
时间窗: Assessed every 3 months for up to 84 months
Patients will be screened and undergo a series of growth measurements on Day 1 and every 3 months thereafter. No study drug is administered.
次要结局
未报告次要终点
研究者
研究点 (20)
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