A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 10
- 主要终点
- Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE)
研究概览
简要总结
The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement therapy (ERT).
Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.
详细描述
The drug being tested in this study is called Velaglucerase Alfa (VPRIV). VPRIV will be tested to treat participants with type 1 Gaucher disease. This study will look at the safety, efficacy and pharmacokinetics of VPRIV in the treatment of type 1 Gaucher disease.
The study will enroll approximately 20 participants with Gaucher disease. The study comprises a screening period (Day -21 through Day -4), baseline period (Day -3 through Day 0), treatment period (Week 1 to Week 51), and safety follow-up period. Participants will be assigned to the following drug administration:
• Velaglucerase Alfa (VPRIV)
Participants will receive VPRIV as intravenous (IV) infusion every other week from Week 1 through Week 51 during the Treatment Period. Percentage of participants with at least one serious treatment-emergent adverse event (TEAE) will be evaluated throughout the study.
This multi-center trial will be conducted in China. The overall time to participate in this study is approximately 59 weeks. Participants will make a safety follow-up telephone call or visit to the site after 30 (±7) days of the last infusion of the study drug.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Velaglucerase Alfa (VPRIV)
Participants received VPRIV IV infusion at 60 units per kilogram (U/kg) body weight once every other week (EOW) for 60 (+10) minutes for up to 51 weeks.
干预措施: Velaglucerase Alfa (Drug)
结局指标
主要结局
Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE)
时间窗: Up to 56.2 weeks
Adverse event(AE)=any untoward medical occurrence in clinical investigation participant administered a drug;it does not necessarily have to have causal relationship with this treatment. AE can therefore be any unfavorable\&unintended sign (example,clinically significant abnormal laboratory value),symptom/disease temporally associated with use of drug whether or not it is considered related to drug. TEAE=any event emerging or manifesting at or after initiation of investigational product or any existing event that worsens in either intensity or frequency following exposure to investigational product. SAE=any untoward clinical manifestation of signs, symptoms, or outcomes(whether considered related to investigational product or not)\&at any dose: results in death,is life-threatening,requires in-patient hospitalization/prolongation of hospitalization,results in persistent/significant disability/incapacity,results in congenital abnormality/birth defect,or is an important medical event.
次要结局
- Percentage of Participants With TEAEs(Up to 56.2 weeks)
- Percentage of Participants With Infusion-related Reactions Reported as an Adverse Event(Up to 56.2 weeks)
- Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53(Week 53)
- Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53(Week 53)
- Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis(Week 53)
- Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53(Week 53)
- Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Measurements at Week 53(Week 53)
- Change From Baseline to Week 53 in Hemoglobin Concentration(Baseline, Week 53)
- Change From Baseline to Week 53 in Platelet Count(Baseline, Week 53)
- Change From Baseline to Week 53 in Normalized Liver Volume(Baseline, Week 53)
- Change From Baseline to Week 53 in Normalized Spleen Volume(Baseline, Week 53)
- Change From Baseline to Week 53 in 36-item Short Form General Health Survey (SF-36) Scores(Baseline, Week 53)
- Change From Baseline to Week 53 in Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50) Scores(Baseline, Week 53)
- Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1(Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1)
- Serum Concentration for VPRIV at Week 37(Within 3 minutes prior to the end of the 60-minute infusion at Week 37)
- Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV(Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1)
- AUCinf: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for VPRIV(Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1)
- Terminal Phase Elimination Half-life (T1/2) for VPRIV(Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1)
- Oral Clearance (CL) for VPRIV(Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1)
- Apparent Steady-state Volume of Distribution (Vss) for VPRIV(Pre-dose and at multiple timepoints(up to 120 minutes post-dose on Day 1 of Week 1)
- Percent Change From Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C Motif] Ligand 18(Baseline, Week 53)
- Percent Change From Baseline to Week 53 in Biomarker: Glucopsychosine(Baseline, Week 53)