A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia

BioMarin Pharmaceutical Inc.0 个研究点目标入组 119 人开始时间: 2018年6月15日最近更新: 2020年11月23日

适应症

试验速览

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•1. Must have completed Study 111-301
•2. 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 research-related procedure. Also, subjects under the age of majority are willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority.
•3. 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
•4. If sexually active, are willing to use contraception as specified in section 9.3.3 of the protocol,
•5. Are willing and able to perform all study procedures
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 119
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Permanently discontinued BMN 111 or placebo prior to completion of Study 111-301
•2. Have a clinically significant finding or arrhythmia on Baseline electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
•3. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays
•4.Require any investigational agent prior to completion of study period
•5.Current therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, GnRH agonists, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function (Table 9.3.2.1)
•6.Planned or expected to have limb-lengthening surgery during the study period
•7.Pregnant or breastfeeding at the Baseline Visit or planning to become pregnant (self or partner) at any time during the study
•8.Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
•9.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

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