Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
2018年5月8日 更新者:BioMarin Pharmaceutical
A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
研究概览
研究类型
介入性
注册 (实际的)
22
阶段
- 阶段2
- 阶段1
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习地点
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Rheinland-pfalz
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Mainz、Rheinland-pfalz、德国、55131
- Zentrum für Kinder- und Jugenmedizin
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Nice、法国、06202
- Hôpital de I´Archet- Centre Hospitalier Universitaire Nice
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Paris Cedex 13、法国、75651
- Hopital Pitie-Salpetriere
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Adelaide, SA
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Adelaide、Adelaide, SA、澳大利亚、5006
- Royal Adelaide Hospital, SA Pathology
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California
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La Jolla、California、美国、92103-8765
- Univ of California San Diego School of Medicine
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Florida
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Gainesville、Florida、美国、32610
- University of Florida College of Medicine
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Kansas
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Kansas City、Kansas、美国、66160
- University of Kansas Medical Center
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Birmingham、英国、B15 2TH
- Old Queen Elizabeth Hospital, Department of Medicine
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London、英国、NW3 2QG
- Royal Free Hospital
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Salford、英国、M6 8HD
- Salford Royal Hospital NHS Trust
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参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
13年 及以上 (孩子、成人、年长者)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion criteria:
- Patient has been diagnosed with Pompe Disease prior to or during the screening period based on 2 GAA gene mutations and either: endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed in cultured skin fibroblasts -or- endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed by dried blood spot or whole blood assay;
- Patient is male or female and 13 years of age or older at the time of enrollment in the study;
- Sexually active patients must be willing to use an acceptable method of contraception while participating in the study and for at least 4 months following the last dose of BMN 701;
- If patient is female and not considered to be of childbearing potential, she is at least 2 years post-menopausal or had tubal ligation at least 1 year prior to screening, or who have had total hysterectomy;
- If patient is female and of childbearing potential, she has negative urine pregnancy tests during the Screening Period and at the Baseline visit and be willing to have additional pregnancy tests during the study;
- Patient has ≥30% predicted upright FVC and either <80% predicted upright FVC, or >10% reduction in supine FVC compared to upright FVC during the Screening Period;
- Patient is naïve to Enzyme Replacement Therapy (ERT) with rhGAA;
- Patient must be able to ambulate at least 40 meters (131.2 feet) on the 6MWT conducted at the Screening visit (use of assistive devices such as walker, cane, or crutches, is permitted); and
- If subject was female, she was not lactating
Exclusion criteria:
- Patient has a history of diabetes or other disease known to cause hypoglycemia and is currently receiving, or might anticipate receiving, hypoglycemic agents during the course of the study;
- Patient has been on any immunosuppressive medication other than glucocorticosteroids within 1 year prior to enrollment into this study;
- Patient requires invasive ventilatory assistance at the time of enrollment into the study;
- Patient has received any investigational medication within 30 days prior to the first dose of study drug or is scheduled to receive any investigational drug other than BMN 701 during the course of the study;
- Patient has previously been admitted to the study;
- Patient is breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
- Patient has a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient's ability to comply with the protocol requirements or compromise the patient's well being or safety;
- Patient has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的:BMN 701
IV infusion
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GILT 标记的重组人 GAA
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Number of Participants With Adverse Events
大体时间:24 weeks
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Number of Participants with Adverse Events as a Measure of Safety and Tolerability
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24 weeks
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
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Change From Baseline in Six Minutes Walk Test
大体时间:Baseline up to 24 weeks
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Change from Baseline in Six Minutes Walk Test.
The 6MWT measured the maximum distance the subject could walk on a flat, hard surface in a period of 6 minutes
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Baseline up to 24 weeks
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其他结果措施
结果测量 |
措施说明 |
大体时间 |
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Change From Baseline in Percent Predicted Upright Forced Vital Capacity
大体时间:Baseline up to 24 week
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Change from Baseline in Percent Predicted Upright Forced Vital Capacity.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 week
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Change From Baseline in Percent Predicted Supine Forced Vital Capacity
大体时间:Baseline up to 24 weeks
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Change from Baseline in Percent Predicted Supine Forced Vital Capacity.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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Change From Baseline in Percent Predicted Upright Maximum Expiratory Pressure
大体时间:Baseline up to 24 weeks
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Change from Baseline in Percent Predicted Upright Maximum Expiratory Pressure.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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Change From Baseline in Percent Predicted Upright Maximum Inspiratory Pressure
大体时间:Baseline up to 24 weeks
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Change from Baseline in Percent Predicted Upright Maximum Inspiratory Pressure.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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Change From Baseline in Upright Maximum Ventilatory Volume
大体时间:Baseline up to 24 weeks
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Change from Baseline in Upright Maximum Ventilatory Volume.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
调查人员
- 研究主任:Medical Monitor、BioMarin Pharmaceutical
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2011年1月17日
初级完成 (实际的)
2013年3月6日
研究完成 (实际的)
2013年3月6日
研究注册日期
首次提交
2010年10月27日
首先提交符合 QC 标准的
2010年10月28日
首次发布 (估计)
2010年10月29日
研究记录更新
最后更新发布 (实际的)
2018年6月11日
上次提交的符合 QC 标准的更新
2018年5月8日
最后验证
2018年5月1日
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- POM-001
- 2010-023561-22 (EudraCT编号)
药物和器械信息、研究文件
研究美国 FDA 监管的药品
是的
研究美国 FDA 监管的设备产品
不
此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.
庞贝病的临床试验
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BioMarin Pharmaceutical终止未接受或接受 rhGAA 治疗的迟发性 Pompe 患者西班牙, 斯洛文尼亚, 塞尔维亚, 大韩民国, 希腊, 澳大利亚, 新加坡, 巴西, 加拿大, 台湾, 爱尔兰, 波兰, 罗马尼亚
BMN 701的临床试验
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BioMarin Pharmaceutical终止
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Terns, Inc.尚未招聘