33 results
The main objective of the project is to study difference between compliance by pill count after 3 months by newly diagnosed multiple myeloma (NDMM) patients with an innovative e-coach, in order to improve treatment compliance by the intervention…
To study the safety and efficacy of deferasirox as treatment of oxidative stress in adult subjects with sickle cell disease.
The Sponsor is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB). This study aims to collect prospective data to characterise bleeding events and…
To study the effect of crizanlizumab on the hemodynamics of the cerebral vasculature (CBF and CVR)
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.
The objectives of this study are twofold, (1) to investigate whether tranexamic acid reduces the volume of blood loss and number of allogenic transfusion, and (2) to investigate the extend of fibrinolysis during burn excisional surgery.
Study AG348-C-018 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical efficacy and safety of mitapivat in subjects with α- or β-TDT. The primary objective of the study is to compare the…
The purpose of this study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed the treatment extension period (without tapering down) of the Phase 2…
To evaluate the prevalence of C-TMA in patients presenting with TMA, either with coexisting conditions or not. Furthermore, (i) the diagnostic performance of an in-house developed ex vivo test, (ii) dynamics of complement measures during follow-up,…
The primary objective of this study is:- To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA)The secondary objectives of this study are:-To compare the proportion of subjects with wAIHA…
- To assess platelet (dys)function in adults with 22q11.2DS and its association with bleeding history.- To identify alterations in metabolic pathways in 22q11.2DS that may be of relevance to mechanisms underlying neurodevelopmental and…
The primary objective of this study is to determine if left atrial appendage closure with the WATCHMAN FLX device is a reasonablealternative to non-vitamin K oral anticoagulants (NOACs) in patients with non-valvular atrial fibrillation.
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
Dose escalation phase:Primary• Determine the RP2D and if reached, the MTD of GEN3014• Evaluate the safety and tolerability of GEN3014 Secondary:• Characterize the PK properties of GEN3014• Characterize the pharmacodynamic properties of GEN3014•…
Primary (Efficacy):To evaluate the efficacy of magrolimab + azacitidine compared with that of azacitidine + placebo in previously untreated participants with intermediate/high/very high risk MDS by IPSS-R as measured by CR rateTo evaluate the…
Primarily, to investigate whether recipient immunity is preserved and how fast it reconstitutes after non-myeloablative MSD HSCT resulting in mixed chimerism in adult SCD patients. Secondly, to investigate whether donor immunity is transferred to…
We aim to determine the long-term integrity of hematopoiesis in pediatric and young adult HSCT survivors.
Primary Objectives: • To evaluate the safety and tolerability of ascending doses of KER 047 in participants with iron-refractory iron deficiency anemia (IRIDA)Secondary Objectives:• To evaluate the pharmacodynamic (PD) effects of KER 047 on iron…
-To evaluate the long-term safety of BIVV001 in previously treated patients with hemophilia A.-To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
This study is being carried out to investigate the treatment with benralizumab in patients with active HES.The purpose of this study is to see if benralizumab, given as injections under the skin, can help control your HES better if it is added to…