An Open-label Extension Study of Subcutaneously Administered Fitusiran in Patients with Moderate or Severe Hemophilia A or B who have Participated in a Previous Clinical Study with Fitusiran

Genzyme Corporation0 个研究点目标入组 48 人开始时间: 2015年7月17日最近更新: 2019年2月28日

研究概览

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•Completed and tolerated study drug dosing in study ALN-AT3SC-001.
•Male aged =18 years.
•Moderate or severe, clinically stable hemophilia A or B as evidenced by a laboratory FVIII or FIX level =5% at screening. Patients with a FVIII or FIX level >5% at screening will be eligible on provision of a historic laboratory report indicating a trough level =5%.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 44
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 4

•Liver disease defined as clinically significant cirrhosis as determined by the Investigator, INR >1.5 at Screening, ALT/AST >3× upper limit of the normal reference range (ULN), platelet count =120,000/mL and/or other complete blood count test results that are considered clinically significant and unacceptable by the Investigator, or known hepatitis C virus currently requiring treatment with ribavirin or interferon.
•Patients known to be human immunodeficiency virus seropositive and have a CD4 count <200 cells/µL at screening.
•History of venous thromboembolism.
•Current serious mental illness that, in the judgment of the Investigator, may compromise patient safety, ability to participate in all study assessments, or study integrity.
•Uncontrolled hypertension.
•Clinically relevant history or presence of cardiovascular, respiratory, gastrointestinal, renal, neurological, inflammatory, or other diseases that, in the judgment of the Investigator, precludes study participation.
•If using nonsteroidal anti-inflammatory drugs intermittently or chronically, must tolerate them with no previous side effects.

发起方