NCT01124643已完成3 期
An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 35
- 试验地点
- 9
- 主要终点
- Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
研究概览
简要总结
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
详细描述
HGT-REP-060 was designed to provide participants who participated in TKT028 an additional 52 weeks of treatment with Replagal at the standard dose of 0.2 milligram per kilogram (mg/kg) every other week (EOW) to assess the effect of continued Replagal therapy on long-term safety and clinical outcomes for this participant population.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Complete all study requirements and assessments for Study TKT028 less than 30 days (+/- 7 days) prior to the first dose in this extension protocol
- •Voluntarily signed an Institutional Review Board/Independent Ethics Committee- approved informed consent form after all relevant aspects of the study have been explained and discussed.
- •Has received and tolerated at least 80% of the total planned Replagal infusions in Study TKT028
- •Female participants of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have a negative pregnancy test at the time of study entry and as required throughout participation in study
排除标准
- •Has received treatment with any investigational drug (other than Replagal) or device within 30 days prior to study entry.
- •Is unable to comply with the protocol, (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator
- •Has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C(HCV) antibody, or human immunodeficiency virus (HIV) antibody
- •Is pregnant or lactating
- •Is morbidly obese, defined as body mass index (BMI) >39 kg/m2
- •Has any safety or medical issues, as assessed by the Investigator, that contraindicate participation in the study (eg, has experienced an adverse reaction to treatment with Replagal or has a known hypersensitivity to any of the components of Replagal
研究组 & 干预措施
Replagal 0.2 mg/kg EOW
Experimental
Intravenous, 0.2mg/kg EOW
干预措施: Replagal (Biological)
结局指标
主要结局
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
时间窗: Baseline to 12 months
Safety Evaluations
时间窗: Baseline to 12 months
次要结局
- Change From Baseline in Albumin/Creatinine (A/Cr) Ratio(Baseline to 12 months)
- Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise(Baseline to 12 months)
- Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)(Baseline to 12 months)
- Change From Baseline in New York Heart Association (NYHA) Functional Class(Baseline to 12 months)
- Change From Baseline in Plasma Gb3(Baseline to 12 months)
- Change From Baseline in eGFR(Baseline to 12 months)
- Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)(Baseline to 12 months)
研究者
研究点 (9)
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