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.
ovartis Healthcare Pvt Ltd0 个研究点目标入组 0 人开始时间: 待定最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
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
研究者
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