EUCTR2017-000701-21-DE进行中（未招募）1 期
A Multicenter, Multinational Clinical Assessment Study for PediatricPatients with Achondroplasia
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 500
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Individuals eligible to participate in this study must meet all of the following criteria:
- •1. 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.
- •2. Birth to = 17 years of age at study entry.
- •3. Have ACH, documented by clinical diagnosis
- •4. Are ambulatory and able to stand without assistance (not applicable for children who are younger than 5 years of age and less than 104 cm in length)
- •5. Are willing and able to perform all study procedures as physically possible
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 500
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Individuals who meet any of the following exclusion criteria are not
- •eligible to participate in the study:
- •1. Have hypochondroplasia or short stature condition other than ACH
- •(e.g., trisomy 21, pseudoachondroplasia)
- •2. Have any of the following disorders:
- •o Hypothyroidism
- •o Insulin-requiring diabetes mellitus
- •o Autoimmune inflammatory disease (including celiac disease, lupus
- •(SLE), juvenile dermatomyositis, scleroderma, and others)
- •o Inflammatory bowel disease
- •o Autonomic neuropathy
- •3. Have an unstable clinical condition likely to lead to intervention during
- •the course ofthe study, including progressive cervical medullary
- •compression
- •4. Growth plates have fused
- •5. Have a history of any of the following:
- •o Renal insufficiency
- •6. Have a history of cardiac or vascular disease, including the following:
- •o Cardiac dysfunction
- •o Hypertrophic cardiomyopathy
- •o Congenital heart disease
- •o Cerebrovascular disease, aortic insufficiency
- •o Clinically significant atrial or ventricular arrhythmias
- •7. Current treatment with antihypertensive medications, ACE inhibitors,
- •angiotensin IIreceptor blockers, diuretics, beta-blockers, calciumchannel
- •blockers, cardiacglycosides, systemic anticholinergic agents, any
- •medication that may impair orenhance compensatory tachycardia, drugs
- •known to alter renal function that isexpected to continue for the
- •duration of the study
- •8. Have been treated with growth hormone, insulin-like growth factor 1
- •(IGF-1), oranabolic steroids in the previous 6 months or long-term
- •treatment (> 3 months) at anytime
- •9. Have had regular long-term treatment (> 1 month) with oral
- •corticosteroids (low-dose ongoing inhaled steroid for asthma is
- •acceptable) in the previous 12 months
- •10. Concomitant medication that prolongs the QT/QTc interval within 14
- •days or5 half-lives, whichever is longer, before the Screening visit
- •11. Have used any other investigational product or investigational
- •medical device for the treatment of ACH or short stature
- •12. Planned or expected bone-related surgery (ie. surgery involving
- •disruption of bone cortex), during the study period. Subjects with
- •previous bone-related surgery may enroll if surgery occurred at least 12
- •months prior to the study and healing is complete without sequelae.
- •13. Planned or expected to have limb-lengthening 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.
- •14. Have any condition that, in the view of the Investigator, places the
- •subject at high risk of poor compliance with the visit schedule or of not
- •completing the study.
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