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Quantification of leukocyte dynamics in bone marrow of healthy humans

To quantify leukocyte turnover, characterize immune phenotype and function, determine T-cell receptor repertoire, and measure soluble immune factors in bone marrow and blood of healthy adults.

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II

This study has been transitioned to CTIS with ID 2023-505904-41-00 check the CTIS register for the current data. Primary Objective: To assess the safety of long-term administration of KVD900 in adolescent and adult patients with HAE type I or II.…

Heavy water labeling of leukocytes in human skin and vet and additional cell populations in fat such as macrophages and adipocytes

The primary objective of this study is to quantify the dynamic properties of different leukocyte subpopulations in healthy skin and blood. Secondly, we aim to understand the relationship between leukocytes in skin and those circulating through the…

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

This study has been transitioned to CTIS with ID 2024-515025-28-00 check the CTIS register for the current data. To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in…

Effectiveness and cost-effectiveness of lymphaticovenous anastomosis for cancer patients who suffer from chronic peripheral lymphedema

Primary Objective:The primary objective is to assess the efficacy of LVA surgery in comparison to sham procedure in patients with cancer related lymphedema in the context of improvement of Lymph-ICF score at 12 and 24 months follow up. Secondary…

The DRAIHA study: Data Registry of AutoImmune Hemolytic Anemia, to improve diagnostic testing for the development of personalized treatment protocols in AIHA patients

Primary objective1. The primary aim of the study is to answer the question whether specification of a positive direct antiglobulin test and/or red blood cell autoantibody specification is correlated with the clinical course in patients with AIHA. We…

National study of inherited platelet function disorders in the Netherlands

Primary objective: to register and investigate Dutch patients suspect for an inherited platelet function disorder, to assess clinical presentation, bleeding score, treatment, burden of disease and quality of life. Secondary objectives: to…

A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO ASSESS THE EFFICACY AND SAFETY OF BIVV009 IN PATIENTS WITH PRIMARY COLD AGGLUTININ DISEASE WITHOUT A RECENT HISTORY OF BLOOD TRANSFUSION

Primary Objective:The purpose of Part A is to determine whether sutimlimab administration results in a greater than or equal to (>=)1.5 gram per deciliter (g/dL) increase in hemoglobin (Hgb) level and avoidance of transfusion in participants…

A prospective, natural history study to assess the occurrence of HPA-1a alloimmunization in women
identified at higher risk for Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

To inform the frequency of women at higher FNAIT risk among pregnant women of different racial and ethnic characteristics who present for pre-natal care and to assess the occurrence of HPA-1a alloimmunization in these women. It is planned that data…

An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria

The objectives of the study are to assess safety and tolerability, pharmacokinetics (PK), and biological activity (including efficacy and pharmacodynamics [PD]) of multiple subcutaneous doses of pegcetacoplan in pediatric patients with paroxysmal…

The sequestration pattern of thrombocytes in healthy adults: results of the ITP liver and spleen scan.

The main objective of this study is to describe the sequestration pattern of platelets in healthy adult volunteers. The secondary objective is to make a comparison between the sequestration patterns in healthy volunteers, and the patterns in ITP…

Efficacy and Safety of M281 in Adults with Warm Autoimmune Hemolytic Anemia: A Multicenter, Randomized, Double blind, Placebo controlled Study with a Long-term Open-label Extension

This study has been transitioned to CTIS with ID 2023-505321-14-00 check the CTIS register for the current data. Primary Objective:Efficacy of nipocalimab in participants with warm autoimmune hemolytic anemia (wAIHA)The key secondary objectives of…

Evaluation of safety and efficacy of mitapivat sulfate in adult patients with sickle cell disease

This study has been transitioned to CTIS with ID 2024-515569-32-00 check the CTIS register for the current data. To study the safety and efficacy of mitapivat in the treatment of adult subjects with sickle cell disease.

Investigating red blood cell deformability of sickle cell patients who started therapy.

Investigating red blood cell deformability changes, during treatment, measured with hyperoxia-hypoxia ektacytometry in sickle cell anemia patients, patients with HbSC disease and patients with HbS-beta-thalassemia.

Impact of Alemtuzumab exposure on immune reconstitution, autoimmunity, risk of infection, chimerism and Graft-versus-Host Disease in children with non-malignant diseases undergoing allogeneic stem cell transplantation. ARTIC International Multicenter Observational Study.

Primary objectiveTo evaluate current clinical practice and develop a population based Campath® (Alemtuzumab) pharmacokinetics model for predicting total Alemtuzumab exposure after i.v. administration before allogeneic stem cell transplantation in…

A Phase 2 Study of Daratumumab Subcutaneous (Dara-SC) Administration in Combination with Carfilzomib and Dexamethasone (DKd) Compared with Carfilzomib and Dexamethasone (Kd) in Participants with Multiple Myeloma who have been Previously Treated with Daratumumab to Evaluate Daratumumab Retreatment

The purpose of this study is to compare the efficacy (rate of very good partial response [VGPR] or better as best response as defined by the International Myeloma Working Group [IMWG] criteria) of daratumumab subcutaneous (Dara-SC) in combination…

Tranexamic acid during excisional burn surgery

This study has been transitioned to CTIS with ID 2024-513573-49-00 check the CTIS register for the current data. The objectives of this study are twofold, (1) to investigate whether tranexamic acid reduces the volume of blood loss and number of…

Von Willebrand disease in the Netherlands - Prospective study (WiN-Pro)

Our primary aim is to investigate the current bleeding tendency of patients with VWD.

Efficacy and Safety of human apotransferrin in patients with β-thalassemia intermedia

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…

An Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients with Advanced Systemic Mastocytosis

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…