ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX - LTE15174

Genzyme Corp.0 个研究点目标入组 281 人开始时间: 2024年2月29日最近更新: 2024年7月22日

研究概览

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•01. Participant must be at least 12 years of age inclusive, at the time of signing the informed consent, 02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial, 03. Male, 04. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. In countries where legal age of majority is above 18 years,a specific ICF must also be signed by the participant's legally authorized representative.

•01. Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis, 02. Current participation in tolerance induction treatment (ITI), 03. Current use of factor concentrates or BPAs as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes. However, participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period, 04. Use of compounds other than factor concentrates or BPAs for hemophilia treatment, 05. Current or prior participation in a gene therapy trial, 06. ALT and/or AST >1.5× upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST >5× ULN for patients who were in the fitusiran arm in the parent study., 07Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort: ? - Clinically significant liver disease ? - History of arterial or venous thromboembolism??

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