2022-502148-10-00已完成3 期
A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 17
- 主要终点
- Measuring change in hemoglobin levels before start of iptacopan treatment and mean of hemoglobin levels at Day 126 and Day 168
研究概览
简要总结
The main question this trial is designed to answer: is iptacopan treatment equally effective to standard of care when switching treatment to iptacopan?
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Signed informed consent must be obtained prior to participation in the study
- •Male and female participants ≥ 18 years of age, at the time of ICF signatures and with a diagnosis of PNH confirmed by PNH clone size WBCs ≥ 10% based on historical data.
- •Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumab or i.v. ravulizumab) for at least 6 months prior to screening
- •Mean hemoglobin level ≥10 g/dL, documented by the mean of all available Hb assessments (minimum 2 measurements) over a period of 6 months before screening visit, collected at any laboratory. In addition to fulfill the Hb eligibility criterion, participants must have two different samples collected during the screening period and tested by the central laboratory with the mean >10 g/dL, prior to starting iptacopan.
- •Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine are to be given according to local regulations, at least 2 weeks prior to first dosing. However, administration of these vaccines less than 2 weeks prior to start of iptacopan treatment is at the discretion of the investigator. If iptacopan treatment is started less than 2 weeks post-vaccination, participant must be given prophylactic antibiotic at the start of iptacopan and for at least 2 weeks after vaccination.
- •If not received previously, vaccination against Haemophilus influenzae infections is recommended, if available and according to local regulations. The vaccines should be given at least 2 weeks prior to initiation of iptacopan treatment. However, administration of these vaccines less than 2 weeks prior to start of iptacopan treatment, is at the discretion of the investigator. If iptacopan treatment is started less than 2 weeks post-vaccination, participant must be given prophylactic antibiotic at the start of iptacopan and for at least 2 weeks after vaccination.
- •Able to communicate well with the investigator, to understand and comply with the requirements of the study
排除标准
- •Participation in any other investigational drug trial or use of other investigational drugs at the time of enrollment
- •Ongoing drug or alcohol abuse that could interfere with patient's participation in the trial.
- •Any medical condition deemed likely to interfere with the patient's participation in the study
- •Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
- •Patients requiring red blood cell transfusion in the 6 months prior to screening or during screening
- •History of stem cell transplantation or any solid organ transplantation
- •Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to study drug administration
- •Presence of fever ≥ 38.0 °C (100.4 °F) within 7 days prior to study drug administration
- •Human immunodeficiency virus (HIV) infection (known history of HIV or test positive for HIV antibody at Screening)
- •A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus
- •Unstable medical condition including, but not limited to, myocardial ischemia, active gastrointestinal bleeding, coexisting chronic anemia unrelated to PNH, or unstable thrombotic event not amenable to active treatment as judged by the investigator at Screening.
- •History of cancer of any part of the body within the past 5 years
结局指标
主要结局
Measuring change in hemoglobin levels before start of iptacopan treatment and mean of hemoglobin levels at Day 126 and Day 168
Measuring change in hemoglobin levels before start of iptacopan treatment and mean of hemoglobin levels at Day 126 and Day 168
次要结局
- Measuring change in hemoglobin levels before start of iptacopan treatment and mean of hemoglobin levels at Day 126 and Day 168
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG
研究点 (17)
Loading locations...
相似试验
招募中
3 期
Study to assess the pharmacokinetics, safety, and tolerability of iptacopan in pediatric PNH patients2024-515926-10-00Novartis Pharma AG5
招募中
2 期
A randomized, controlled study to evaluate LNP023 (iptacopan) in patients with active ANCA-associated vasculitis.2023-510525-15-00Novartis Pharma AG44
招募中
4 期
A rollover extension program (REP) to evaluate the long-term safety and tolerability of open label iptacopan in adult participants with primary IgA nephropathy2023-508690-92-00Novartis Pharma AG100
已完成
不适用
A Study to Evaluate the Efficacy of a Facial Sunscreen in Adult Participants With Oily Skin and Signs of AgingNCT07310706Kenvue Brands LLC73
招募中
3 期
Prevention of thrombotic events in the context of long distance flights2024-515630-33-00Klinikum der Universitaet Muenchen AöR27,698