EUCTR2020-004438-39-IT进行中（未招募）1 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
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 81
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
- •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 × 109 cells/L (= 100,000 cells/µL; = 100 G/L).
- •c. Absolute neutrophil count of = 0.75 × 109 cells/L (= 750 cells/µL; = 0.75 × G/L).
- •d. Platelet count of = 30 × 109/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 = 50 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
- •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 75
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 6
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.
- •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 supplementation with an unstable dose in the 28 days prior to the screening visit.
- •10. Clinically significant abnormal electrocardiogram (ECG) at the screening visit.
- •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.
- •13. Positive serology for human immunodeficiency virus, or active infection with hepatitis B virus or hepatitis C virus, unless receiving antiviral therapy and viral load is undetectable.
- •14. Positive drugs of abuse screen, unless by prescription.
- •15. Pregnant, planning to become pregnant, or breastfeeding.
- •16. Known hypersensitivity to BCX9930 or any of its formulation excipients.
- •17. History of severe hypersensitivity to any medicinal product, which was associated with swelling, severe rash requiring treatment/hospitalization, or anaphylaxis.
- •18. Any other clinically significant medical or psychiatric condition that, in the opinion of the investigator or sponsor, would interfere with the subject's ability to participate in the study or increase the risk of participation for that subject.
研究者
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