NCT01435772终止2 期
A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 21
- 试验地点
- 24
- 主要终点
- Number of Participants With a Positive Anti-BMN 701 Antibody
研究概览
简要总结
This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 13 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have completed a prior BMN 701 clinical development study;
- •Have provided written informed consent after the nature of the study has been explained prior to performance of any study-related procedures. Minors may participate as long as they provide written assent after the nature of the study has been explained to them and after their parent, or legal guardian has provided written informed consent, prior to the performance of any study-related procedures;
- •Have been diagnosed with late-onset Pompe Disease, based on the entry criteria of a prior BMN 701 study;
- •If sexually active, be willing to use 2 known effective methods of contraception from Screening until 4 months after the last dose of study-drug;
- •If female, and not considered to be of childbearing potential, be at least 2 years post-menopausal, or have had tubal ligation at least 1 year prior to screening, or have had a total hysterectomy;
- •If female, and of childbearing potential, have a negative pregnancy test during the Screening Period and at the Baseline visit, and be willing to have additional pregnancy tests during the study;
- •Have the ability to comply with the protocol requirements, in the opinion of the Investigator.
排除标准
- •Have received any experimental or approved therapy for Pompe disease, other than BMN 701, subsequent to completion of a BMN 701 study and prior to entry into POM-002;
- •Have received, or are anticipated to receive, any investigational medication, other than BMN 701, within 30 days prior to the first dose of study-drug;
- •Are breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
- •Have 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.
研究组 & 干预措施
BMN 701 10mg/kg
Experimental
BMN 701 10mg/kg IV every other week
干预措施: BMN 701 (Biological)
BMN 701 20mg/kg
Experimental
BMN 701 20mg/kg IV every other week
干预措施: BMN 701 (Biological)
BMN 701 5mg/kg
Experimental
BMN 701 5mg/kg IV every other week
干预措施: BMN 701 (Biological)
结局指标
主要结局
Number of Participants With a Positive Anti-BMN 701 Antibody
时间窗: Baseline, Week 144
Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples.
Number of Participants With a Positive Anti-BMN 701 Antibody Response
时间窗: Baseline, Week 144
Status of Anti-IGF-I antibody is corresponding to the test results of blood samples
次要结局
- Plasma IGF-I Concentration(Baseline, Week 144)
- Change From Baseline in Urine Tetrasaccharide Concentration at Week 144(Baseline, Week 144)
- Percent Predicted Maximal Inspiratory Pressure (MIP)(Baseline, Week 144)
- 6 Minutes Walk Test (Meters)(Baseline, Week 144)
- Percent Predicted Upright Forced Vital Capacity (FVC)(Baseline, Week 144)
- Percent Predicted Maximum Expiratory Pressure (MEP)(Baseline, Week 144)
- Insulin-like Growth Factor Binding Protein 3 (IGFBP3)(Baseline, Week 144)
- Maximum Voluntary Ventilation (MVV)(Baseline, Week 144)
- Plasma IGF-II Concentration(Baseline, Week 144)
研究者
研究点 (24)
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