NCT06100900已完成1 期
An Open-Label, Multicenter, Intra-Subject Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Therapeutic Potential of BCX10013 in Subjects with Paroxysmal Nocturnal Hemoglobinuria
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings.
研究概览
简要总结
This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH. Approximately 8 participants will be enrolled in this study. Participants may receive treatment for up to 52 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or non-pregnant, non-lactating female adults ≥ 18 years old.
- •Documented diagnosis of PNH confirmed by flow cytometry.
- •Body mass index (BMI) ≤ 40 kg/m^
- •Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with ravulizumab for at least 12 months prior to the screening visit and have received no treatment with eculizumab or pegcetacoplan for 6 months prior to the screening visit.
- •Documentation of current vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae type B [Hib] or willingness to start vaccination series at least 14 days prior to Day
排除标准
- •Known history of or existing diagnosis of hereditary complement deficiency.
- •History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
- •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.
- •History of malignancy within 5 years prior to the screening visit.
- •Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
- •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.
- •Receiving iron with an unstable dose (ie, increasing or decreasing) in the 28 days prior to the screening visit.
研究组 & 干预措施
BCX10013
Experimental
Participants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur.
干预措施: BCX10013 (Drug)
结局指标
主要结局
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings.
时间窗: up to 52 weeks
次要结局
- CFB in Lactate Dehydrogenase(Baseline, Week 52)
- CFB in the Ratio of Total PNH Red Blood Cell Clone Size to PNH White Blood Cell Clone Size(Baseline, Week 52)
- CFB in Hemoglobin(Baseline, Week 52)
- Percentage of Participants who are Transfusion-free(52 weeks)
- Percentage of Participants Achieving a Within-subject Clinically Meaningful CFB in the FACIT-Fatigue scale(52 weeks)
- CFB in Other Clinical Biomarkers of PNH Disease Activity including absolute reticulocyte count, total PNH red blood cell clone size, haptoglobin levels, total bilirubin, and aspartate transaminase(Baseline, Week 52)
- Number of Participants with Clinical PNH Symptoms(up to 52 weeks)
- Concentration of BCX10013 and its Metabolite(s) in Plasma(Pre-dose, 0.5, 1, 2, 4, and 6 hours post dose on Day 1, Week 2, and Week 4)
- Concentration of BCX10013 and its Metabolite(s) in Urine (if applicable)(Pre-dose and all urine from 0 to 6 hours post dose on Day 1, Week 2, and Week 4)
研究者
研究点 (1)
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