ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX

Genzyme Corporation0 个研究点目标入组 54 人开始时间: 2017年5月31日最近更新: 2019年6月30日

研究概览

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•Males =12 years of age.
•Severe hemophilia A or B with inhibitors evidenced by:
•a.A central laboratory measurement or documented medical record evidence of FVIII <1% or FIX level =2% at Screening.
•b.On-demand use of BPAs to manage bleeding episodes for at least the last 6 months prior to Screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria:
•Inhibitor titer of =0.6 BU/mL at Screening, or
•Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers =0.6 BU/mL, or
•Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of anamnestic response
•A minimum of 6 bleeding episodes requiring BPA treatment within the last 6 months prior to Screening.
•Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of patients under the age of legal consent, per local and national requirements.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 5
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 46
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 3

•Patients with known co-existing bleeding disorders other than hemophilia A or B
•Patients with clinically significant liver disease
•Patients known to be HIV positive and have a CD4 count <200 cells/µL
•Patients with a history of arterial or venous thromboembolism
•Estimated glomerular filtration rate =45 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula)
•Patients with a co-existing thrombophilic disorder
•Patients with a history of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
•Patients with a history of intolerance to SC injection(s)
•Patients with an anticipated or planned need for surgery during the study
•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 judgement

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