A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 65
- 试验地点
- 10
- 主要终点
- Number of participants with abnormal safety laboratory parameters, vital signs and ECGs
研究概览
简要总结
This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
详细描述
The extension study Baseline/Day 1 visit is equivalent to the End of Treatment visit of the parent study. The study will begin on Day 1 followed by on-site visits every 4 months during the study treatment period. A Safety Follow Up tele-visit must be conducted 7 days after last study treatment to collect information on Adverse Events.
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 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent must be obtained prior to participation in the open label extension study
- •Willing and able to comply with the study Schedule of Activities
- •Participants who have completed the full study treatment period of any prior "Novartis sponsored" iptacopan Phase 3 clinical trial in aHUS (e.g. CLNP023F12301 , CLNP023F12302), are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement
- •Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e., any boosters required should be administered according to local guidelines)
排除标准
- •Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
- •Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk
- •Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- •History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
- •Pregnant or nursing (lactating) women
- •Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping of investigational drug.
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Iptacopan 200 mg
Open label , single arm
干预措施: Iptcaopan 200 mg (Drug)
结局指标
主要结局
Number of participants with abnormal safety laboratory parameters, vital signs and ECGs
时间窗: Throughout the study duration, up to 4 years
Number of participants with abnormal safety laboratory parameters , vital signs and ECGs will be provided
Number of participants with adverse events and serious adverse events
时间窗: Throughout the study duration, up to 4 years
Number of participants with adverse events and serious adverse events, including abnormal safety laboratory parameters, vital signs and ECGs
次要结局
- Number of participants with absence of aHUS relapse without the use of anti-C5 antibody(Throughout the study duration, up to 4 years)
- Number of participants with complete TMA response status without the use of anti-C5 antibody therapy(Throughout the study duration, up to 4 years)
- Estimated glomerular filtration rate (eGFR)(Throughout study duration, up to 4 years)
- Chronic kidney disease (CKD) stage(Throughout study duration, up to 4 years)
- Number of participants by dialysis requirement status(Throughout the study duration, up to 4 years)
- Number of participants with Thrombotic Microangiopathy (TMA) related adverse events(Throughout study duration, up to 4 years)