ATLAS-PPX trial: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis. - ATLAS-PPX

ALNYLAM PHARMACEUTICALS, INC.0 个研究点目标入组 90 人开始时间: 2018年10月19日最近更新: 2021年7月26日

研究概览

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•- Males =12 years of age.
•- Severe hemophilia A or B (evidenced by a central laboratory FVIII <1% or FIX level =2% at Screening) with or without inhibitors
•- At least 1 bleeding episode in the last 12 months
•- AT activity =60% at Screening
•- Documented details of prophylactic treatment with factor concentrates or bypassing agents for the treatment of hemophilia A or B for at least 6 months
•- 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
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 1
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 78
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 5

•- 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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