A Multicenter Open-label Study of the Safety and Efficacy of α-galactosidase A (R-h α-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 6
- 试验地点
- 7
- 主要终点
- Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
研究概览
简要总结
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease.
Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 64 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria)
- •In the case of male participants, documented plasma or leukocyte alpha-galactosidase A (α-GAL) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants)
- •Left ventricular hypertrophy was noted.
- •Accumulation of globotriaosylceramide (GL-3) in the myocardium or a genetic deficiency associated with α-GAL was confirmed
- •Or in the case of heterozygous female participants, when the family (father or son) was diagnosed with Fabry disease. (Father or son was related by birth.)
- •Without symptoms or signs of Fabry, such as acroparesthesia, angiokeratomas, abnormal sweating, pain of distal extremities, chronic abdominal pain/diarrhea and corneal opacities were observed, except for proteinuria sign.
- •Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent
- •Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent.
- •Participants classification: inpatients and outpatients
- •Participants who had given written informed consent before the study-related baseline tests.
排除标准
- •Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury [mmHg] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication)
- •Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent.
- •Participants who had undergone kidney transplantation or were currently on dialysis.
- •Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value five times as high as the upper normal limit).
- •Permanent pacemaker or defibrillator implanted participants
- •Pregnant or lactating women
- •Participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent.
- •Participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent.
- •Enzyme replacement therapy history, except for agalsidase beta
- •Participants who were unwilling to comply with the requirements of the protocol.
- •Others judged by the investigator or sub-investigator to be ineligible for the study
研究组 & 干预措施
Agalsidase Beta
Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.
干预措施: Agalsidase beta (Drug)
结局指标
主要结局
Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
时间窗: Baseline to Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
时间窗: Baseline to Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156
时间窗: Baseline to Week 156
Left ventricular mass was assessed by echocardiogram.
Change From Baseline in LVM at Week 156
时间窗: Baseline to Week 156
Left ventricular mass was assessed by echocardiogram.
次要结局
- Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test(Baseline to Week 156)
- Percent Change From Baseline in GL-3 Plasma Levels at Week 156(Baseline to Week 156)
- Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156(Baseline to Week 156)