NL-OMON51609撤回2 期
A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM-1
适应症
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 4
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
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Male or female, aged >= 18 years old.
- •2. Body weight >= 40 kg.
- •3. Documented diagnosis of PNH confirmed by flow cytometry with a PNH
- •granulocyte or monocyte clone size of >= 10% during screening.
- •4. Treated with a stable regimen of eculizumab for >= 3 months prior to the
- •screening visit or ravulizumab for >= 6 months prior to the screening visit.
- •5. Recorded the following results during screening:
- •a. Hb of <= 105 g/L (<= 10.5 g/dL).
- •b. ARC of >= 100 × 10^9 cells/L (>= 100,000 cells/µL; >= 100 G/L).
- •c. Absolute neutrophil count of >= 0.75 × 10^9 cells/L (>= 750 cells/µL; >= 0.75 ×
- •d. Platelet count of >= 30 × 10^9/L (>= 30,000/µL; >= 30 G/L).
- •e. Adequate iron reserve based on ferritin >= LLN or total iron binding capacity
- •<= upper limit of the normal reference range (ULN).
- •f. Estimated glomerular filtration rate of >= 60 mL/min/1.73 m2 using the
- •Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (Levey and
- •Stevens 2010).
- •6. Contraception requirements: WOCBP and partners of male subjects to use
- •highly effective contraception
- •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.
- •(Note: Vaccination for N. meningitidis type B and for H. influenzae type B
- •(Hib) is strongly encouraged where authorized and available.)
- •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
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
- •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 supplementation with an unstable dose in the 28 days prior to
- •the screening visit.
- •10. Clinically significant abnormal electrocardiogram (ECG) at the screening
- •(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. Aspartate aminotransferase (AST; also serum glutamic-oxaloacetic
- •transaminase [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 as detailed in Section 9.8.1.
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