A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 10
- 试验地点
- 5
- 主要终点
- Assess the safety and tolerability of a single dose of TT30.
研究概览
简要总结
The purpose of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients with PNH.
详细描述
This is an open-label, single-dose, dose-escalation study to assess the safety, tolerability, PK, PD, and immunogenicity of TT30 given as an IV infusion and as a SC injection in subjects with PNH or evidence of circulating PNH cells.
Eligible subjects with PNH will be vaccinated with meningococcal vaccine at least two weeks prior to dosing (if not previously vaccinated or if revaccination is required).
On study day 1, the subject will receive the single dose of TT30 as an IV infusion over 60 minutes or as a SC injection, and be followed with additional study evaluations over the next 59 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 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
- •Individuals at least 18 years of age with a diagnosis of PNH & vaccination against meningococcus.
排除标准
- •Abnormal renal or liver function
- •History of meningococcal disease
- •History of Guillain-Barre syndrome
- •Known infection with HIV or Hepatitis B or C
- •History of thrombotic events
研究组 & 干预措施
TT30 (ALXN1102 Formulation)
IV: 0.1, 0.3, and 1.0 mg/kg
干预措施: ALXN1102 (Drug)
TT30 (ALXN1103 Formulation)
IV: 3.0, 6.0, and 10.0 mg/kg
SC: 1.0 and 3.0 mg/kg
干预措施: ALXN1103 (Drug)
结局指标
主要结局
Assess the safety and tolerability of a single dose of TT30.
时间窗: 60 days
Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.
次要结局
- Characterize PK, PD and immunogenicity of a single dose of TT30.(60 days)