CTRI/2021/03/032089Other1 期
Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog alfa (activated) in Treatment of Episodic Bleeding in Subjects with Inherited Bleeding Disorders.
试验速览
- 阶段
- 1 期
- 状态
- Other
- 入组人数
- 8
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
入选标准
- •Study candidates must meet all the following inclusion criteria to be eligible for participation in this study:
- •1) Confirmed diagnosis of cohort:
- •a) Confirmed diagnosis of congential Factor VII deficiency (FVIID)
- •b) Confirmed diagnosis of congenital Glanzmann thrombasthenia (GT) (ie, platelet function analyzer, mutational analysis)
- •c) Confirmed diagnosis of congenital Hemophilia A with inhibitors on emicizumab (HAwI-E) treated with the same dose of emicizumab
- •2) History of bleeding with an (a) Annualized bleeding rate (ABR) of ââ?°Â¥8 for FVIID. (b) Annualized bleeding rate (ABR) of ââ?°Â¥8 for GT. (c) Annualized bleeding rate (ABR) of ââ?°Â¥1 for HAwI-E
- •3) Agreement to use highly effective birth control throughout the study if the subject has childbearing potential
- •4) 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 during Screening.
- •5) Subjectââ?¬•s ability to rapidly assess a bleeding episode and respond appropriately
- •6) Affirmation of informed consent with signature confirmation and assent for children from age 12 to 17 years before any study-related activities
- •7) Subjectââ?¬•s ability to administer MarzAA SC at home
排除标准
- •Subjects who meet any of the following criteria will not be eligible for participation in this study:
- •1) Cohort 1: genotype of FVIID subjects with following mutations:
- •a) P.A354V-p.464Hfs
- •b) P.Ser112-Stop (homozygous)
- •c) Ala294Val + Del C
- •d) 100GLN ARG shift
- •e) Ser103 Gly
- •Note: documentation of historic genotype would be acceptable.
- •2) Inability to discontinue and washout any prophylactic (except Hemlibra) or episodic treatment for 5 days and 10 days for platelet transfusion prior to dosing
- •3) Previous participation in a clinical study involving SC administration of wt-rFVIIa (NovoSeven or MOD-5014) or any study using a modified amino-acid sequence FVIIa (other than MarzAA) such as: NN1731 or BAY86-6150.
- •Note: Prior participation in a study of intravenous (IV) LR769, rFVIIa-FP (CSL689), or MarzAA is permissible.
- •4) Previous participation in a clinical study with treatment within the previous 30 days or ââ?°Â¤5 half-lives (of the investigation product) or absence of clinical effect, whichever is longer
- •5) Known positive antibody to FVIIa or variants thereof detected during screening or prior to Day 1
- •6) Known hypersensitivity to pd-FVIIa, pd-FVII, wt-rFVIIa, or MarzAA or any of the excipients or related products
- •7) Treatment with anticoagulants or antiplatelet therapy within 1 week of enrollment or anticipated need during the study
- •8) Planned elective surgery within 12 months following study entry
- •9) History of clinically relevant coagulation blood disorders
- •10) CD 4 T cell count of <200 cells/mm3
- •11) Platelet count <50,000 /Ã?¼L based on screening laboratory assessments
- •12) Current or history of advanced atherosclerotic disease (ie, known history of coronary artery disease, ischemic stroke, etc), or deep venous thrombosis (DVT) within 24 months of dosing or considered to be at a high risk of venous thromboembolic event (VTE) or pulmonary embolism as judged by the Investigator
- •13) Compromised hepatic or renal function: a) Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels ââ?°Â¥5 Ã?â?? the upper limit of normal (ULN)
- •b) Total bilirubin level ââ?°Â¥2 mg/dL ( >35 Ã?¼mol/L) unless there is a known history of Gilbertââ?¬•s syndrome
- •c) Serum creatinine level >1.25 Ã?â?? ULN
- •14) 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
- •15) Weight ââ?°Â¥105 kg (231 lbs)
研究者
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