A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 40
- 试验地点
- 18
- 主要终点
- PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo
研究概览
简要总结
This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.
详细描述
Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Type 1 Gaucher disease, the most common form accounts for greater than (>) 90% of cases and does not involve the central nervous system (CNS). Typical manifestations of Type 1 Gaucher disease include splenomegaly, hepatomegaly, thrombocytopenia, anemia, skeletal pathology and decreased quality of life. The disease manifestations are caused by the accumulations of glucosylceramide (storage material) in Gaucher cells which have infiltrated the spleen and liver as well as other tissue. Eliglustat tartrate is a small molecule developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells.
This study was designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate in adult participants (>16 years) with Gaucher disease Type 1. The study consisted of 2 periods: The Double-Blind Primary Analysis Period (PAP [Day 1 to Week 39]) and the Long Term Treatment Period (LTTP/Open-Label Period (post-Week 39 [Day 1 of the Open-Label Period] through study completion).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant (and/or their parent/legal guardian) was willing and able to provide signed informed consent prior to any study-related procedures to be performed;
- •The participant was at least 16 years old at the time of randomization;
- •The participant had a confirmed diagnosis of Gaucher disease Type 1;
- •Female participants of childbearing potential must had a documented negative pregnancy test prior to dosing. In addition all female participants of childbearing potential must use a medically accepted form of contraception throughout the study.
排除标准
- •The participant has had a partial or total splenectomy;
- •The participant had received pharmacological chaperones or miglustat within 6 months prior to randomization;
- •The participant had received enzyme replacement therapy within 9 months prior to randomization;
- •The participant had Type 2 or 3 Gaucher disease or was suspected of having Type 3 Gaucher disease;
- •The participant had any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary, neurologic, endocrine, metabolic, (for example, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illness that might confound the study results, or, on the opinion of the investigator, might preclude participation in the study;
- •The participant had tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen;
- •The participant had received an investigational product within 30 days prior to randomization;
- •The participant was pregnant or lactating.
研究组 & 干预措施
Active
Eliglustat
干预措施: Eliglustat tartrate (Drug)
Placebo
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo
时间窗: PAP Baseline (Day 1), Week 39
Percent change in spleen volume = (\[spleen volume at Week 39 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN.
次要结局
- PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39(PAP Baseline (Day 1), Week 39)
- PAP: Percent Change From Baseline in Platelet Counts at Week 39(PAP Baseline (Day 1), Week 39)
- PAP: Hemoglobin Level(PAP Baseline (Day 1))
- LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234(PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234)
- PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39(PAP Baseline (Day 1), Week 39)
- LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234(PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234)
- LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234(PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234)
- LTTP: Percent Change From Baseline in Platelet Counts at Week 234(PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234)