A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Percentage of Participants With Adverse Events (AEs)
研究概览
简要总结
The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).
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
- •Completed participation in Part C of study ALN-AS1-001 (NCT02452372)
- •Not on a scheduled regimen of hemin
- •Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception
- •Willing and able to comply with the study requirements and to provide written informed consent
排除标准
- •Clinically significant abnormal laboratory results
- •Received an investigational agent (other than ALN-AS1) within 90 days before the first dose of study drug or are in follow-up of another clinical study
- •History of multiple drug allergies or intolerance to subcutaneous injection
研究组 & 干预措施
Givosiran
At the beginning of this study, participants received either givosiran 2.5 mg/kg subcutaneous (SC) injection once monthly(QM), givosiran 5.0 mg/kg SC injection QM, or givosiran 5.0 mg/kg SC injection once every 3 months (Q3M). Within a year, all participants were transitioned to givosiran 2.5 mg/kg SC injection QM.
干预措施: Givosiran (Drug)
结局指标
主要结局
Percentage of Participants With Adverse Events (AEs)
时间窗: Through Month 49
An AE is any untoward medical occurrence in a participant or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.
次要结局
- The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) as Measured by Percent Decrease From Baseline(Baseline; Month 48)
- Annualized Rate of Hemin Administration(Through Month 49)
- Annualized Rate of Composite Porphyria Attacks(Through Month 48)
- The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) as Measured by Percent Decrease From Baseline(Baseline; Month 48)