Open-label Study of PTG-300 in Subjects With Hereditary Hemochromatosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Effect of PTG-300 on Transferrin Saturation
研究概览
简要总结
This study will be conducted at multiple sites and every patient will get treated with PTG-300. The objective of the study is to assess the effect of PTG-300 in treating adult hereditary hemochromatosis patients.
详细描述
This study is a multicenter, open-label study. The objective of the study is to assess the effect of PTG-300 on transferrin saturation (TSAT) and serum iron in adult hereditary hemochromatosis patients.
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
- •Men and women aged 18 and older
- •Confirmed diagnosis of HFE-related hereditary hemochromatosis with prior genotype testing
- •Documented stable phlebotomy for ≥ 6 months
- •Screening hemoglobin >11.5 g/dL
- •Documented evidence of prior serum ferritin ≥500 ng/mL
- •Serum ferritin >50 ng/mL and <300 ng/mL at screening
排除标准
- •Clinically meaningful laboratory abnormality
- •Receiving iron chelation therapy
- •Receiving erythrocytapheresis
- •Pregnant or lactating females
- •Infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection within 3 months of dosing; any infections requiring antimicrobial therapy within 4 weeks of dosing.
- •Serious or unstable medical or psychiatric condition that would prevent subject providing informed consent
- •Organ damage from iron overload
- •Primary or secondary immunodeficiency
- •Positive hepatitis B or hepatitis C or known human immunodeficiency virus infection
- •Known history of autoimmune/inflammatory diseases
- •Any surgical procedures requiring general anesthesia within 1 month prior to screening or planned surgery during study
- •History of invasive malignancies within last 2 years, except non-malignant skin cancer and local cured prostate cancer, cervical cancer, or ductal carcinoma in situ
- •Receipt of an investigational agent within 30 days of screening
研究组 & 干预措施
PTG-300
PTG-300 Subcutaneous
干预措施: PTG-300 (Drug)
结局指标
主要结局
Effect of PTG-300 on Transferrin Saturation
时间窗: Week 24 (or End of Treatment if treated for less than 24 weeks)
Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.
Effect of PTG-300 on Serum Iron
时间窗: Week 24 (or End of Treatment if treated for less than 24 weeks)
Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests.
次要结局
- Effect of PTG-300 on Phlebotomy Frequency(Over 24 weeks)