A Phase 3, single-arm, multicenter, multinational, open-label, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged >= 12 years with severe hemophilia A or B with or without inhibitory antibodies to factor VIII or IX

Tanaka Tomoyuki0 个研究点目标入组 75 人开始时间: 2023年5月14日最近更新: 2023年11月20日

适应症

试验速览

暂无简介。

•Diagnosis of severe congenital hemophilia A or B (FVIII <1% or FIX level <=2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence
•- For participants currently not on prophylaxis (clotting factor concentrates [CFC] or bypassing agents [BPA] on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) 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 participants under the age of legal consent, per local and national requirements

•Participants are excluded from the study if any of the following criteria apply:
•- Known co-existing bleeding disorders other than congenital hemophilia A or B
•- History of arterial or venous thromboembolism, not associated with an indwelling venous access.
•- History of intolerance to subcutaneous (SC) injection(s).
•- Current participation in immune tolerance induction therapy (ITI)
•- Prior gene therapy
•- Current or prior participation in a fitusiran trial
•- Current or prior participation in a gene therapy trial
•- Received an investigational drug or device within 30 days prior to the screening visit or within 5 half-lives of the investigational drug (or device) prior to the screening visit, whichever is longer
•- Presence of clinically significant liver disease
•- AT activity <60% at Screening
•- Co-existing thrombophilic disorder
•- Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
•- Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
•- Presence of acute or chronic hepatitis B infection
•- Known to be human immunodeficiency virus (HIV) positive with CD4 count <200 cells/micro L.
•- Reduced renal function

发起方

Tanaka Tomoyuki