EUCTR2020-003172-41-IT进行中（未招募）1 期
A multicenter, single-arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who are naive to complement inhibitor therapy. -
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
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 participants = 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with RBCs and white blood cells (WBCs) (granulocyte/monocyte) clone size = 10%.
- •- Mean hemoglobin level <10 g/dL confirmed by central laboratory assessment during Screening and prior to starting study treatment:
- •a. By two hemoglobin measurements (mean < 10 g/dL), two to eight weeks apart, for patients not receiving a RBC transfusion during Screening.
- •b. By one hemoglobin measurement (<10 g/dL) carried at the first Screening visit for patients receiving a RBC transfusion after which he/she will be eligible.
- •- LDH > 1.5 x Upper Limit of Normal (ULN) for at least two central laboratory measurements two to eight weeks apart during the screening period.
- •- Vaccination against Neisseria meningitidis infection is required prior to the start of 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 dosing.
- •- If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given, if available and according to local regulations. The vaccines should be given at least 2 weeks prior to first dosing.
- •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 36
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •- Prior treatment with a complement inhibitor, including anti-C5 antibody.
- •- Known or suspected hereditary complement deficiency at screening.
- •- History of hematopoietic stem cell transplantation.
- •- Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <500x106/L).
- •- Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to study drug administration.
- •- A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- •- Major concurrent comorbidities including but not limited to severe kidney disease (e.g., dialysis), advanced cardiac disease (e.g., New York Heart Association (NYHA) class IV), severe pulmonary disease (e.g., severe
- •pulmonary hypertension (World Health Organization (WHO) class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.
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