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A Phase 3 Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients with Hereditary Angioedema (HAE)

Primary objective: The primary objective of the study is to evaluate the clinical efficacy of ISIS 721744 in patients with hereditary angioedema (HAE).Secondary objective: Evaluate the effects of ISIS 721744 on the quality and pattern of HAE attacks…

Targeting Pyruvate kinase as a therapeUtic option in rare aneMiA - PUMA-Study

We aim to further explore the role of decreased activity and stability of PK in several types of hereditary hemolytic anemia and non-regenerative anemia. Moreover, we will study the effect of restoring this instability using second generation (…

Sickle Red blood Cell Survival: explaining heterogeneity

To examine the determinants of survival of donor RBCs in SCD patientsTo look into the effect of transfusion on the innate immune system of sickle cell patients, in particular, the phenotype of the neutrophils.

He-move-philia, a combined lifestyle intervention program for patients with hemophilia and other congenital coagulation disorders

The aim of this exploratory study is to compare the effect of a lifestyle intervention program consisting of combined individual coaching and group sessions (CLI) with a lifestyle intervention program with individual coaching only (II).The program…

Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients - The DosEmi study

This study has been transitioned to CTIS with ID 2024-515528-35-00 check the CTIS register for the current data. Primary objectiveTo determine whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is non-inferior to conventional…

Lymphomics; improving the understanding of the anatomy of the lymphatic system and the direction and velocity of lymph flow

To improve the knowledge and understanding of the (patho)physiology of lymph disorders Main objective: the anatomy of the central lymph vessels, the velocity and direction of lymph flow and its influencers and thoracic duct outlet location and…

A clinical study to investigate Interferon gamma (IFN*) signature in patients post HSCT and in patients with impaired HSC proliferation pre-transplant

HSCT cohort:* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the risk of graft failure* -To investigate the relationship between IFNγ levels and IFNγ activity by measuring CXCL9 levels and the…

An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

This study has been transitioned to CTIS with ID 2023-509201-77-00 check the CTIS register for the current data. Primary ObjectiveTo evaluate the safety of long-term dosing with donidalorsen in patients with HAE. Secondary ObjectivesTo evaluate the…