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

Genzyme Corporation0 个研究点目标入组 120 人开始时间: 2018年3月22日最近更新: 2021年11月15日

适应症

试验速览

暂无简介。

•Males =12 years of age. In Denmark only males = 18 years of age.
•Severe hemophilia A or B without 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 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
•A minimum of 6 bleeding episodes requiring factor concentrate 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. Assent is N/A in Denmark as only patients = 18 years of age will be included
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 8
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 77
•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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