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Do Serum Alemtuzumab Concentrations Predict Donor T Cell Chimerism after Non-Myeloablative Matched Sibling Donor Stem Cell Transplantation in Sickle Cell Disease Patients? (PREDICT study)

To investigate whether serum alemtuzumab concentrations are predictive of the robustness of engraftment in SCD patients undergoing a matched sibling donor transplantation with alemtuzumab/TBI conditioning resulting in mixed chimerism.

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 (…

A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy

Part 1 - To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in subjects with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to…

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.

Functional investigation of a variant in ARGHAP18 in a family with mastocytosis

To investigate the functional consequences of the found variant in ARGHAP18 in mast cell behaviour.

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…

A PIVOTAL, OPEN-LABEL, MULTICENTER STUDY TO ASSESS THE EFFICACY AND SAFETY OF BIVV009 IN PATIENTS WITH PRIMARY COLD AGGLUTININ DISEASE WHO HAVE A RECENT HISTORY OF BLOOD TRANSFUSION

Part APrimary Objective:• The primary objective of Part A is to determine whether BIVV009 administration results in a >= 2 g/dL increase in hemoglobin (Hgb) levels or increases Hgb to >= 12 g/dL and obviates the need for blood…

Sickle Cell Disease: Targeting Alloantibody formation Reduction; RIsk factors, aNd Genetics

In this study, the genetic risk factors and time dependent risk factors for alloimmunization will be analyzed. Furthermore, the role of the innate and adaptive immune system on allo-antibody formation in SCD patients will be elucidated.

NeutrOphil acTivity and dIversity (phenotype) in Sickle Cell disEase and their changes after treatment; The NOTICE study

The overall aim of this project is to delineate the role of neutrophil activity and diversity in the pathogenesis of acute (e.g. vaso-occlusive crisis (VOC) and Acute chest syndrome (ACS)) and chronic complications in sickle cell didease and its…

A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

* The primary objective of this study is to compare the proportion of warm antibody autoimmune hemolytic anemia (wAIHA) subjects whoachieve a durable hemoglobin response between the fostamatinib and placebo groups.* The secondary objectives of this…

A Phase 3, Open -label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) versus Epoetin alfa for the Treatment of Anemia due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in ESA Naïve Subjects who require Red Blood Cell Transfusions.

This study has been transitioned to CTIS with ID 2022-501485-22-00 check the CTIS register for the current data. Primary objective:• To evaluate the efficacy of luspatercept on RBC transfusion independence (RBC-TI for 12 weeks [84 days] with an…

Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B

This study has been transitioned to CTIS with ID 2024-510738-42-00 check the CTIS register for the current data. The primary aim of the trial is to demonstrate the non-inferiority of CSL222 (formerly AMT-061) (2 × 1013 gc/kg) during the 52 weeks…

Leukapheresis of healthy volunteer donors for T cell study and TolDC production process validations

To obtain fresh mononuclear cells from healthy volunteer donors to be used for validation of procedures for the generation of GMP-grade T cell or tolerogenic dendritic cell products for clinical application.

Post-traumatic dysfunction of the innate and adaptive immune system and its relationship with the development of infectious complications in severely injured patients (POSEIDON study)

We expect to provide evidence for our hypothesis stating that patients who develop infectious complications after trauma are characterized by a specific subset of circulating neutrophils displaying a reduced capacity to eradicate bacteria.…

A Phase 3, Randomized, Double-blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)

This study has been transitioned to CTIS with ID 2023-510107-22-00 check the CTIS register for the current data. To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.

Sickle Cell Outcome REsearch

To determine the natural history of sickle cell disease and to identify modifying factors, including: (epi)genetic, biological, pathophysiological, social/ demographic, psychological and therapeutic determinants, which contribute to morbidity and…

A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease

* To evaluate the fetal hemoglobin (HbF) response to IMR-687 versus placebo * To evaluate the safety of IMR-687 versus placebo

A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia

This is a phase 2 study intended to explore the potential use of IMR 687 to treat subjects with β thalassemia. This is the first study of IMR-687 in a β-thalassemia population, and, as such, is designed to examine the safety, tolerability, and PK,…

Coagubility in severe mitral regurgitation: A within-patient evaluation of coagulation status before and after Mitral Valve Transcatheter Edge-to-Edge Repair.

To evaluate coagulation status in patients with moderate-severe to severe MR, with concomitant atrial enlargement or atrial fibrillation, before and after M-TEER to explore the complex relationship between MR and coagulability.

Restrictive versus Liberal Thresholds for Red Blood Cell Transfusion in ExtraCorporeal Membrane Oxygenation

The primary objective of this trial is to study in a prospective randomized comparison whether a restrictive RBC transfusions strategy is non-inferior compared to a liberal strategy in patients on ECMO with respect to 90-day mortality.