A multicenter, 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
- 尚未招募
- 发起方
- 入组人数
- 100
- 试验地点
- 5
- 主要终点
- Is iptacopan safe, tolerable and effective in participants with aHUS
研究概览
简要总结
The purpose of this Phase 3b study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in participants with atypical hemolytic uremic syndrome (aHUS). All participants should have successfully completed the full study treatment period in a Novartis-sponsored Phase 3 study of iptacopan in aHUS.
aHUS is a very rare disease that happens when there is an uncontrolled activation of an important system in your body called the alternative complement pathway (AP). The AP usually provides protection against infections. However, in aHUS, the over-activation of AP can lead to destruction of red blood cells, low platelets and damage small blood vessels in your kidneys causing inflammation. This is known as thrombotic microangiopathy (TMA). The constant inflammation causes continued damage to your kidneys.
Iptacopan is a new treatment tested in aHUS. It is believed that it may improve aHUS by reducing inflammation and helping your kidneys. Iptacopan acts on a protein called factor B in your immune system. Inhibition of factor B has the potential to prevent the destruction of red blood cells by your own immune system resulting in maintenance of normal hemoglobin levels.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 18.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Approximately 100 male and female participants, ≥18 years of age, with a diagnosis of aHUS prior to screening.
- •Participants must have completed the full study treatment period of a Novartis sponsored iptacopan Phase 3 clinical trial in aHUS, are still on iptacopan study treatment and derive benefit from it as per Investigators judgement.
- •Already vaccinated against bacteria such as Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae.
- •If the patient has not been previously vaccinated, or if a booster is required, vaccine will be given if available and according to local regulations, at least 2 weeks prior to first dosing of iptacopan.
排除标准
- •Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the drugs listed in Section 6.8.2
- •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.
结局指标
主要结局
Is iptacopan safe, tolerable and effective in participants with aHUS
时间窗: Vitals, adverse events and laboratory parameters are followed in all visits since day 1 to end of tretment visit. | ECG is carried out on Day 1, Month 12,24,36,48 till End of traetment To be performed once in a year (i.e. at Month 12, Month 24, Month 36, Month 48 etc. until the EoT visit)
次要结局
- To evaluate the proportion of participants who were free of aHUS signs and symptoms at time of enrollment and do not have these return during their participation in the study(Absences of aHUS signs and symptoms without the use of anti C5 antibody treatment are carried out in all visits since day 1 to end of study treatment.)
研究者
Murugananthan K
Novartis Healthcare Private Limited