CONSENT II: Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Measurement of Serum Lactate Dehydrogenase (LDH)
研究概览
简要总结
Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.
详细描述
Coversin, a small protein complement C5 inhibitor which prevents the cleavage of C5 by C5 convertase into C5a and C5b, will be used in an open label, non-comparative clinical trial in patients with PNH and proven resistance to eculizumab due to C5 polymorphisms. Patients will be treated with Coversin by daily subcutaneous injection for 6 months in order to determine the safety and efficacy of the drug in these circumstances. If satisfactory control of the PNH is achieved, and at the discretion of the Principal Investigator (PI), patients will have the option of remaining on Coversin and being entered into the long term follow-up study.
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
- •Patients with known PNH.
- •Aged 18 and above. No upper age limit.
- •Lactate dehydrogenase (LDH) ≥1.5 upper limit of normal.
- •Must agree to use two methods of contraception that are ≥99% effective in preventing pregnancy.
- •Resistance to eculizumab (Soliris®).
- •Voluntary written informed consent.
- •Willing to self-inject Coversin daily.
- •Willing to receive appropriate prophylaxis against Neisseria infection.
- •Willing to avoid prohibited medications for duration of study.
排除标准
- •Subjects with body weight <50 kg (110 lb) or >100 kg (220 lb).
- •Pregnancy or breast feeding (females).
- •Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom).
- •Unresolved Neisseria meningitidis infection.
- •Patients who have not received adequate immunization against Neisseria meningitides.
- •Impaired hepatic function.
- •Patients with impaired renal function.
- •Failure to satisfy the Principal Investigator (PI) of fitness to participate for any other reason.
研究组 & 干预措施
Coversin treatment
Coversin - 22.5mg followed by 45mg for 6 months.
干预措施: Coversin (Drug)
结局指标
主要结局
Measurement of Serum Lactate Dehydrogenase (LDH)
时间窗: Baseline to Day 186
Measurement of serum lactate dehydrogenase (LDH)
次要结局
- Percentage Change in Haemoglobin (Hb) Compared to Screening(Baseline to Day 186)
- Number of Packed Red Blood Cells (PRBC) Transfusions(Baseline to Day 180)