EUCTR2020-005186-13-SI进行中（未招募）1 期
A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult aHUS patients who are naive to complement inhibitor therapy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 50
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Male and female patients = 18 years of age with evidence of thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and kidney injury, based on the following laboratory findings:
- •- Platelet count <150x109/L during the Screening Period or within 28 days prior to the start of the Screening Period, and
- •- 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 with acute worsening of kidney function. (patients requiring dialysis for acute kidney injury are eligible)
- •Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections 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
- •If not received previously, vaccination against Haemophilus influenzae infection should be given if available and according to local regulations. The vaccine should be given at least 2 weeks prior to first study drug administration.
- •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
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 45
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •Treatment with complement inhibitors, including anti-C5 antibody
- •A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) deficiency, and/or Shiga toxin-related hemolytic uremic syndrome (STX-HUS), and/or Positive direct Coombs test
- •Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase e (DGKE) mediated aHUS
- •Started receiving PE/PI 28 days or longer before the start of screening visit for the current TMA
- •Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation
- •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
- •Among patients with native kidney, history or presence of any kidney disease other than aHUS, such as:
- •- Known kidney biopsy finding suggestive of underlying disease other than aHUS
- •- Kidney ultrasound finding demonstrating small kidneys suggestive of chronic kidney failure
- •- Known family history and/or genetic diagnosis of non-complement mediated genetic kidney disease (eg, focal segmental glomerulosclerosis)
- •- Laboratory tests indicative of a kidney disease other than aHUS (eg, Anti-glomerular Basement Membrane (anti-GBM) antibodies, Anti- Neutrophil Cytoplasmic Antibodies (ANCA), Anti-Phospholipase A2 Receptor (anti-PLA2R) antibodies, anti-double stranded DNA (antidsDNA)
- •antibodies)
- •- Liver disease or liver injury at screening
- •- Patients with sepsis, severe systemic infection or Coronavirus Disease 2019 (COVID-19) infection
- •-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
- •Active infection, or history of recurrent invasive infections, caused by encapsulated bacteria (i.e. meningococcus, pneumococcus), or H. influenzae
- •Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
- •Chronic hemo- or peritoneal dialysis
- •Any adenoviral COVID-19 vaccine within 28 days prior to screening visit
研究者
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