Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 15
- 主要终点
- Incidence of change from baseline in blood pressure for rapid infusion-1
研究概览
简要总结
Background: In order to allow our satisfied patients, who have successfully completed 24 months of rapid intravenous infusion of Velaglucerase alfa (VPRIV), to continue with the 10 minutes IV therapy, the clinical trial framework must be extended; and this extension is important for the assessment of long term benefit (up to 5 years) of this regimen of administration of Velaglucerase alfa..
Suggested trial: An additional 36 months home therapy follow up of safety and efficacy of rapid intravenous infusion of Velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease.
Patients must have completed the prior 4 parts / 24 months of the protocol before enrolling into this extension phase ("Part 5") and have provided a new consent before entering PART 5 of the study.
Patients must not have experienced clinically significant AEs, including allergic reactions, in any of the prior study parts of this protocol to be eligible to participate, and have maintained stability in the key disease features.
All infusions of 10' will be given in the context of home therapy. "Clinically significant" AEs will be determined by the PI using standard description of AEs as previously described at phase 3, and if necessary will support withdrawal of the patient from the study.
详细描述
Every 6 months, patients will be required to come for routine checkups at SZMC, where the following tests will be performed:
- Complete Blood Count (CBC)
- Routine serum biochemistry including liver function tests (LFTs)
- Plasma biomarker lyso Gb-1
- Height & weight & calculation of BMI
- Physical examination and medical history elicited including concomitant medications
- Ultrasound for spleen and liver volumes
In addition, the following tests will be performed at 12, 24 and 36 months:
- Echocardiography
- Electrocardiogram (ECG)
- Urinalysis
- HRQoL questionnaire (TBD)
At each home visit, the following assessments will be performed by the study nurse:
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
- 6 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 18-75 years, non-splenectomized Enzymatic diagnosis & molecular analysis indicative of type 1 Gaucher disease Receiving VPRIV for at least 6 infusions (3 months) prior to Baseline at a constant dose and frequency and without clinically significant AEs including allergic reactions
排除标准
- •Experience of a clinically significant AE to VPRIV at any time in the past Existence of a clinically significant co-morbidity
研究组 & 干预措施
Rapid infusion of Vpriv
: Infusions at Baseline and during step-wise rate increases and End-of-study will be performed in the Shaare Zedek Medical Center (SZMC) by the Study Nurse who will monitor vital signs (see below) for a total of 8 visits at SZMC. Home therapy will be approved if the patient so desires for 5 infusions in Phase 1 and for the first 5 infusions in Phase 3. All routine hematological and biochemical tests will be performed in the SZMC clinical labs. Abdominal quantitative MR Imaging (MRI) for spleen and liver volumes will be performed at SZMC
干预措施: VPRIV (Drug)
结局指标
主要结局
Incidence of change from baseline in blood pressure for rapid infusion-1
时间窗: 9 months
measured by blood pressure pre and post infusion
Incidence of change from baseline in heart rate for rapid infusion-1
时间窗: 9 months
measured by heart rate pre and post infusion
Incidence of change from baseline in temperature for rapid infusion-1
时间窗: 9 months
measured by temperature pre and post infusion
次要结局
- Non deterioration in Gaucher manifestations- measured by lack of elevated biomarker- Lyso-GB1(9 months)
- Non deterioration in Gaucher manifestations- measured by spleen volumes(9 months)
- Non deterioration in Gaucher manifestations- stability in platelet counts(9 months)
- Non deterioration in Gaucher manifestations- stability in hemaglobin count(9 months)
- Non deterioration in Gaucher manifestations- measured by liver volume(9 months)
研究者
Ari Zimran
Professor
Shaare Zedek Medical Center