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A Phase 3 open-label, multicenter study of the safety, efficacy, and pharmacokinetics of intravenous recombinant coagulation Factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated pediatric patients <12 years of age with severe hemophilia A

To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A.

The Effect of Voxelotor on Cerebral Perfusion and Oxygenation (ESR-C005)

To study the effect of voxelotor on the hemodynamics of the cerebral vasculature (CBF and CVR)

A 2-Part, Phase 1, Multicenter, Single Dose, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CSL889 in Adult Patients with Sickle Cell Disease

Primary Objective:To assess the safety and tolerability of single doses of CSL889 administered by IV infusion in subjects with stable SCD and in subjects with SCD in VOCSecondary Objectives:1. To characterize the PK profile of CSL889 after single IV…

Blood Outgrowth Endothelial Cells (BOECs) and Megakaryocytes (MKs) for in vitro studies of hemostatic and secretory function.

1. To establish optimized approaches for the isolation and characterization of BOECs, CD34+-derived MKs, iPSC- ECs and iPSC-MKs.2. Dissect the cellular mechanisms that control endothelial and platelet (secretory) function, thereby understanding the…

An Open-Label Extension Study of ISIS 721744 in Patients with Hereditary Angioedema

This study has been transitioned to CTIS with ID 2024-517249-15-00 check the CTIS register for the current data. Primary Objective:To evaluate the safety of extended dosing, and alternative dosing and/or dose frequency with ISIS 721744 in patients…

A Phase I/IIb extension study assessing the long-term safety and efficacy of an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.

This study has been transitioned to CTIS with ID 2024-512603-39-00 check the CTIS register for the current data. Primary: To assess the long-term safety (6-10 years after dosing) of a systemic administration of AAV5-hFIX, an AAV vector containing a…