ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX

Sanofi K.K.0 个研究点目标入组 120 人开始时间: 2018年11月5日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

研究类型: Interventional
分配方式: Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment

•Inclusion Criteria:
•1. Males, >=12 years of age
•2. Severe hemophilia A or B without inhibitors
•- Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is <=2% at Screening;
•- On-demand use of factor concentrate to manage bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion:
•* Nijmegen modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening
•* No use of BPAs to treat bleeding episodes for at least the last 6 months prior to Screening
•* No history of immune tolerance induction therapy within the last 3 years prior to Screening
•3. A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening.
•4. Willing and able to comply with the study requirements and to provide written informed consent and assent

•Exclusion Criteria:
•- Known co-existing bleeding disorders other than hemophilia A or B, ie, Von Willebrand's disease, additional factor deficiencies, or platelet disorders.
•- Antithrombin (AT) activity <60% at Screening
•- Co-existing thrombophilic disorder
•- Clinically significant liver disease
•- Active HCV infection
•- HIV positive with a CD4 count of <200 cells/microL
•- History of arterial or venous thromboembolism
•- Inadequate renal function
•- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
•- History of intolerance to SC injection(s)
•- Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment

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