CTRI/2021/10/037662招募中3 期
A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 (Iptacopan) in adult atypical hemolytic uremic syndrome (aHUS) patients who are naive to complement inhibitor therapy.
Novartis Healthcare Pvt Ltd8 个研究点 分布在 1 个国家目标入组 50 人开始时间: 2021年11月18日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 8
- 主要终点
- The primary objective (performed at the end of the Core Treatment period (i.e the first 26 weeks) is: To assess the proportion of participants treated with iptacopan achieving complete thrombotic microangiopathy (TMA) response during 26 weeks of study treatment.
研究概览
简要总结
The study is designed as a multicenter, single-arm, open label study todemonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mgb.i.d. in adult patients with aHUS who are treatment naive to complementinhibitor therapy (including anti-C5 antibody). The study will assess theeffects of iptacopan on a range of efficacy assessments relevant to aHUSincluding hematological and kidney parameters, dialysis requirement, changes inchronic kidney disease (CKD) stage, as well as patient reported outcomes (PRO)for fatigue and quality of life. This study will serve as the pivotal trial forthe development of iptacopan as a treatment for patients with aHUS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 盲法
- Open Label
入排标准
- 年龄范围
- 18.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •1-Male and female patients ≥ 18 years of age with evidence of thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury, based on the following laboratory findings: 2-Platelet count <150x109/L during the Screening Period or within 28 days prior to the start of the Screening Period, and 3-LDH ≥1.5 x upper limit of normal (ULN) during the Screening Period or within 28 days prior to the start of the Screening Period and hemoglobin ≤ lower limit of normal (LLN) for age and gender during the Screening Period or within 28 days prior to the start of the Screening Period, and •Serum creatinine ≥ULN during the Screening Period (patients requiring dialysis for acute kidney injury are eligible) •Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment.
- •If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local regulations, at least 2 weeks prior to first study drug administration.
- •If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination 4-If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given according to local regulations.
- •The vaccines should be given at least 2 weeks prior to first study drug administration.
- •If iptacopan study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study medication and for at least 2 weeks after vaccination.
- •5-Among patients with a kidney transplant, (a) known history of aHUS prior to current kidney transplantation, or (b) no known history of aHUS, and persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen.
排除标准
- •1-Treatment with complement inhibitors, including anti-C5 antibody 2-ADAMTS13 deficiency, and/or Shiga toxin-related hemolytic uremic syndrome (STX-HUS), and/or Positive direct Coombs test 3-Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS 4-Receiving PE/PI, for 28 days or longer, prior to the start of screening for the current TMA 5-Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation 6-In patients with a kidney transplant, acute kidney dysfunction consistent with the diagnosis of transplantation failure due to acute/chronic active T-Cell mediated rejection (TCMR) and/or active/chronic active antibody-mediated rejection (ABMR) according to Banff 2017 criteria 7-Among patients with native kidney, history of any kidney disease other than aHUS, such as: 8-Known kidney biopsy finding suggestive of underlying disease other than aHUS 9-Kidney ultrasound finding demonstrating small kidneys suggestive of chronic kidney failure 10-Known family history and/or genetic diagnosis of non-complement mediated genetic kidney disease (eg, focal segmental glomerulosclerosis) 11-Liver disease or liver injury at screening 12-Patients with sepsis, severe systemic infection or COVID-19 infection 13-Presence of systemic infections (bacterial, viral, fungal or parasitic) that, in the opinion of the Investigator, confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease 14-Active infection, or history of recurrent invasive infections, caused by encapsulated bacteria (i.e. meningococcus, pneumococcus), or H.
- •influenzae 15-Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome 16-Chronic hemo- or peritoneal dialysis.
结局指标
主要结局
The primary objective (performed at the end of the Core Treatment period (i.e the first 26 weeks) is: To assess the proportion of participants treated with iptacopan achieving complete thrombotic microangiopathy (TMA) response during 26 weeks of study treatment.
时间窗: End of Week 26
次要结局
- To assess the effect of iptacopan on-(-time to complete TMA response)
研究者
研究点 (8)
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