NCT03381833终止2 期
A Multi-Center, Randomized, Open-Label, Parallel-Group Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 84
- 试验地点
- 1
- 主要终点
- Effect of LJPC-401 on cardiac iron
研究概览
简要总结
This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with transfusion-dependent beta thalassemia with myocardial iron overload.
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
- None
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients ≥ 14 years of age with transfusion-dependent beta thalassemia.
- •Patients must have increased iron levels in the heart as measured by magnetic resonance imaging (MRI). Two separate cardiac T2*MRI from 6 to 35 msec; <= 15% difference between the two.
- •Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable dose prior to study and expected to remain stable during study.
- •Female patients of childbearing potential must not be pregnant, must have negative pregnancy tests, and must use an effective birth control method during the study.
- •Male patients must be either surgically sterile or use an effective birth control method during the study.
- •Patient must be willing and able to provide written informed consent. Parent of legal guardian to patients younger than age of majority must be willing and able to provide informed consent.
排除标准
- •Any significant medical condition or lab abnormality that would prevent the patient from participating in the study.
- •Pregnant or lactating women.
- •Patients taking an immunosuppressive agent (except topical over-the-counter steroids, inhaled steroid medications, and non-steroidal anti-inflammatory drugs) or have a planned surgery (except dental surgery or simple dermatologic procedures).
- •Patients participating in an unapproved investigational clinical trial within 30 days of this study.
- •Patients with a disease, disability or condition which may interfere with the conduct of the study, or which would, in the opinion of the Investigator, pose an unacceptable risk to the patient.
- •Patients who are unwilling or unable to comply with the study requirements.
- •Patients with known hepatitis B or hepatitis C, or being treated for a positive viral load or are noncompliant with hepatitis medications.
- •Known and active human immunodeficiency virus (HIV) infection.
- •Patients with Child Pugh class C cirrhosis or liver failure.
- •Patients with severe congestive heart failure (NYHA Class 4).
- •Use of erythropoiesis stimulating agents in the past 3 months prior to study entry.
- •History of allergic reaction to hepcidin or excipients.
- •Contraindication to MRI scanning.
研究组 & 干预措施
Group A - Delayed therapy
Active Comparator
standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks
干预措施: LJPC-401 (Drug)
Group B - Immediate therapy
Active Comparator
standard chelation therapy plus LJPC-401 for 52 weeks
干预措施: LJPC-401 (Drug)
结局指标
主要结局
Effect of LJPC-401 on cardiac iron
时间窗: 26 Weeks
Change in cardiac T2\* magnetic resonance imaging (MRI)
次要结局
- Effect of LJPC-401 on cardiac iron(52 Weeks)
- Effect of LJPC-401 on hepatic iron(52 Weeks)
- Effect of LJPC-401 on serum iron(56 Weeks)
- Effect of LJPC-401 on hemoglobin(52 Weeks)
- Effect of LJPC-401 on volume of blood transfused(56 Weeks)
- Effect of LJPC-401 on its potential to elicit an immune response(56 Weeks)
- Effect of LJPC-401 on the incidence of treatment-emergent adverse events(56 Weeks)
- Effect of LJPC-401 on vital signs-heart rate(56 Weeks)
- Effect of LJPC-401 on vital signs-body temperature(56 Weeks)
- Effect of LJPC-401 on vital signs-respiratory rate(56 Weeks)
- Effect of LJPC-401 on vital signs-blood pressure(56 Weeks)
- Effect of LJPC-401 on body weight(56 Weeks)
研究者
研究点 (1)
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