38 results
1.1 Overall Aim: To establish a safe, standardized protocol for the perioperative management of patients with atrial fibrillation (AF) who are receiving a novel oral anticoagulant (NOAC) drug, either dabigatran, rivaroxaban or apixaban, and require…
The ATLAS-A/B trial (ALN-AT3SC-004) is a multicenter, multinational, randomized, openlabel Phase 3 study designed to demonstrate the efficacy and safety of fitusiran in patients with hemophilia A or B without inhibitory antibodies to FVIII or FIX…
1.3. Study Design Rationale The ATLAS-INH trial (ALN-AT3SC-003) is a multicenter, multinational, randomized, openlabel Phase 3 study designed to demonstrate the efficacy and safety of fitusiran in patients with haemophilia A or B with inhibitory…
Is it possible to detect prothrombotic states via the "calibrated automated thrombogram" in patients that endured CVST, who did no show deviations in the prothrombin time/activated partial thromboplastin time? Secondary, we will…
Objective: Primary objective: Red blood cell aggregation and elongation (deformability) assessed by a laser assisted optical rotational cell analyzer (LORCA) on days 1-3, and within 24 hours after weaning of ECLS. Secondary objectives are red blood…
Primary objective:To investigate the safety of turoctocog alfa pegol during continuous use for prevention and treatment of bleeding episodes of previously turoctocog alfa pegol treated severe haemophilia A patients.Secondary objectives:To…
The primary objectives of this study are to establish the efficacy and safety of APL-2 compared to eculizumab in patients with PNH who continue to have Hb levels <10.5 g/dL despite treatment with eculizumab.
1. To assess the effect of increased hematocrit on cerebral perfusion as depicted by CBF and CVR.2. To determine the effect of GBT440 on oxygen saturation of the cerebral vasculature and the oxygen local consumption.3. Determine the dose response…
1.3. Study Design Rationale The ATLAS-PPX trial (ALN-AT3SC-009) is a multicenter, multinational, open-label Phase 3 switching study designed to demonstrate the efficacy and safety of fitusiran in patients with hemophilia A or B, with inhibitory…
The main objective of the current study is to investigate platelet reactivity and underlying mechanisms using the novel multi-microspot analysis in a group of patients with SIHD and a healthy control group.
The primary objective of this randomized controlled trial is to study the effectiveness of lymphaticovenous anastomosis (LVA) compared to complex decongestive therapy (CDT) in terms of health-related quality of life (HRQoL). Secondary objectives are…
To assess platelet function and coagulation parameters in adult chronic ITP patients in relation to their bleeding phenotype
The objective of this open-label extension (OLE) study is to assess the effect of long-term safety and treatment effect of Voxelotor in participants who have completed treatment in Study GBT440-031, using the following parameters:- Safety based upon…
Primary: • The primary objective is to determine adjudicated ORR (CR/CR with partial recovery of peripheral blood counts [CRh] + PR + CI) based on modified IWG-MRT-ECNM consensus response criteria in patients with AdvSM treated with avapritinib and…
The purpose of this first in human study is to determine if the gene transfer study product, BAY 2599023 (DTX201), is safe and has beneficial effects for treating hemophilia A.
Our primary aim is to investigate the current bleeding tendency of patients with VWD.
To study the effect of apotransferrin administration in patients with β thalassemia intermedia on erythropoiesis as reflected by hemoglobin level or transfusion dependency. Secondary objectives are the effect of apotransferrin on the…
- Identify hemato-oncology patients and conditions with a high versus a low bleeding risk, by epidemiological research and a short questionnaire. - Investigate the association of bleeding related biomarkers with bleeding.
Primary Objective: The main goal of this project is to identify CVR predictors including CBF, age, sex and vascular stressors in anaemic and control patients using several MRI techniques. While anaemia is correlated with other cerebrovascular risk…
- Primary Objectives: To establish a minimum of 6 months of prospective efficacy data of FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for NAb to AAV-Spark100.- Secondary Objectives:…