EUCTR2020-000996-19-BG进行中（未招募）1 期
Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog Alfa (Activated) For On-Demand Treatment and Control of Bleeding Episodes in Subjects with Haemophilia A or Haemophilia B, with Inhibitors: The Crimson 1 Study - The Crimson 1 Study
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 60
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) Confirmed diagnosis of congenital Haemophilia A or B with inhibitors with one of the following:
- •a) Titer of =5BU
- •b) Titer of =0.6 BU but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the subject’s medical history, precluding the use of FVIII or FIX products to treat bleeding as documented by the Investigator
- •c) Titer =0.6 BU but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the subject’s medical history, precluding the use of FVIII or FIX products to treat bleeding as documented by the Investigator
- •Note: Documentation of highest historic titer should be recorded.
- •2) History of bleeding with a historic ABR of =8
- •3) Male or Female, Age =12 years of age
- •4) Agreement to use highly effective birth control throughout the study if the subject has childbearing potential
- •5) If female the subject must meet the following criteria:
- •a) Not currently be breastfeeding
- •b) Not plan on becoming pregnant during the study
- •c) Be surgically sterile, or at least 2-years postmenopausal, or have a negative serum pregnancy test at Screening (Visit 1)
- •6) Affirmation of informed consent with signature confirmation and assent for children between ages 12 to 17 before any study-related activities Note: study-related activities are any procedure that would not have been performed during normal clinical management of the subject
- •7) Stated willingness to comply with all study procedures and availability for the duration of the study
- •8) Investigator confirmed subject’s ability to rapidly assess a bleeding episode and respond appropriately
- •9) Investigator confirmed subjects’ ability to administer MarzAA SC and infuse standard of care at home
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 28
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 32
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Previous participation in a study involving SC administration of rFVIIa (NovoSeven or MOD-5014) or any study using a modified amino-acid sequence FVIIa (other than MarzAA) such as: NN1731 or BAY86-6150. Prior participation in a study of IV LR769, rFVIIa-FP (CSL689), or MarzAA is permissible.
- •2) Previous participation in a clinical study with subsequent treatment within the previous 30 days or =5-half-lives or absence of clinical effect, whichever is longer
- •3) Known positive antibody to FVIIa or variants thereof wt-rFVIIa detected during screening and prior to Day 1
- •4) Known hypersensitivity to pd-FVIIa, pd-FVII, wt-rFVIIa or MarzAA or any of the excipients or related products
- •5) Treatment with anticoagulants or antiplatelet therapy within one week of enrolment or anticipated need during the study
- •6) Planned elective surgery within 12 months following study entry
- •7) History of other clinically relevant coagulation disorders
- •8) Platelet count <50,000 /µL based on screening laboratory assessments
- •9) Current or history of advanced atherosclerotic disease (i.e., known history of coronary artery disease, ischemic stroke, etc.), or deep venous thrombosis (DVT) or pulmonary embolism (PE) within 24 months of dosing or considered to be at a high risk of venous thromboembolic event (VTE) as judged by the Investigator
- •10) CD4 T Cell count <200 cells/mm3
- •11) Compromised hepatic or renal function:
- •a) Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels =5 x the upper limit of normal (ULN)
- •b) Total bilirubin level =2 mg/dL (>35 µmol/L) unless there is a known history of Gilbert’s syndrome
- •c) Serum creatinine (Cr) level >1.25 × ULN
- •12) Inability or medical, psychosocial, or familial issues that might prevent full participation and cooperation with the procedures and requirements of the clinical study as determined by the potential subject and physician/Investigator
- •13) Weight =105 kg (231 lbs)
研究者
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