EUCTR2020-004403-14-CZ进行中（未招募）1 期
A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria - REDEEM 2
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 57
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
- •1.Male or female, aged 18 - 70 years old at screening.
- •2.Body weight = 50 kg.
- •3.Documented diagnosis of PNH confirmed by flow cytometry with a PNH granulocyte or monocyte clone size of = 10% during screening.
- •4.Are either: (a) naïve to treatment with a complement inhibitor, or (b) have received no treatment with a complement inhibitor for at least 12 months prior to the screening visit.
- •5.Recorded the following results during screening:
- •a.Hb = 105 g/L (= 10.5 g/dL).
- •b.LDH = 2 × upper limit of normal reference range (ULN)
- •c.ARC of = 100 × 109 cells/L (= 100,000 cells/µL; = 100 G/L).
- •d.Absolute neutrophil count (ANC) of = 0.75 × 109 cells/L (= 750 cells/µL; = 0.75 G/L).
- •e.Platelet count of = 30 × 109/L (= 30,000/µL; = 30 G/L).
- •f.Estimated glomerular filtration rate of = 50 mL/min/1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (Levey and Stevens 2010).
- •6.Contraception requirements:
- •Female participants must meet at least one of the following requirements:
- •a.Be a woman of nonchildbearing potential.
- •b.Be a woman of childbearing potential who agrees to use a highly effective contraceptive method throughout the study and for a duration of 30 days after the last dose of study drug.
- •c.Alternatively, true abstinence is acceptable for women of childbearing potential when it is in line with the subject’s preferred and usual lifestyle.
- •Male participants must meet at least one of the following requirements:
- •a.Males with a female partner of childbearing potential must use condoms throughout the study and for a duration of 90 days after the dose of study drug unless their partner is using a highly effective contraceptive method independent of the study.
- •b.Alternatively, true abstinence is acceptable when it is in line with the subject’s preferred and usual lifestyle
- •7.Documentation of current vaccinations against Neisseria meningitidis types A, C, W, and Y, and Streptococcus pneumoniae, or willingness to start vaccination series at least 14 days prior to Day 1.
- •8.In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures and restrictions for the study, including compliance with the BID dosing schedule for BCX9930.
- •9.Willing and able to provide written informed consent
- •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 52
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1.Known history of or existing diagnosis of hereditary complement deficiency.
- •2.History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
- •3.Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis.
- •4.History of malignancy within 5 years prior to the screening visit, with exception of adequately treated non-melanoma skin or superficial bladder cancer, curatively treated carcinoma in situ of the cervix, or other curatively treated solid tumor deemed by the investigator and medical monitor to be at low risk for recurrence.
- •5.Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening.
- •(Note: Suspected or confirmed coronavirus disease [COVID-19]; persistent or recurrent positive test(s) for severe acute respiratory syndrome coronavirus 2 [SARS CoV 2] nucleic acids or antigens; and worsening of dyspnea not due to PNH, vasculitic rash, and persistent fever or other symptoms consistent with multisystem inflammatory syndrome in adults [MIS A] are exclusionary.)
- •6.Current participation in any other investigational drug study or participation in an investigational drug study within 30 days prior to the screening visit, or 5.5 half-lives of the investigational drug, whichever is longer.
- •7.Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
- •8.Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit.
- •(Note: Treatment with these medications initiated > 28 days prior to the screening visit is not exclusionary, if the dose is stable and there is a reasonable expectation that treatment will be continued.)
- •9.Receiving iron with an unstable dose in the 28 days prior to the screening visit.
- •10.Clinically significant abnormal electrocardiogram (ECG) at the screening visit.
- •(Note: This includes, but is not limited to, a QT interval corrected using Fridericia’s method [QTcF] of > 450 msec in males or > 470 msec in females, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping.)
- •11.Subjects with any of the following results at the screening visit:
- •a.Alanine aminotransferase (ALT; also serum glutamic-pyruvic transaminase [SGPT]) > 3 × ULN.
- •b.AST (SGOT) > 3 × ULN.
- •(Note: Subjects may be enrolled with AST > 3 × ULN if explained by hemolysis.)
- •c.Total serum bilirubin > 2 × ULN
- •(Note: Subjects may be enrolled with total serum bilirubin > 2 × ULN if explained by hemolysis or Gilbert’s syndrome. In the case of hemolysis, total serum bilirubin must be < 5 × ULN and in the case of Gilbert’s syndrome, total serum bilirubin must be < 11 × ULN.)
- •12.Current use of a prohibited concomitant medication within 7 days prior to Day 1
- •13.Positive serology for human immunodeficiency virus, or active infection with hepatitis B virus or hepatitis C virus.
- •14.Positive drugs of abuse screen, unless by prescription or alcohol dependence, defined as
- •consumption of = 40 g pure alcohol per day for men and = 20 g pure alcohol for women.
- •15.Pregnant, planning to be
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