Optimization of Prime Fluid Strategy to Preserve Microcirculatory Perfusion During Cardiac Surgery...
Endothelial DysfunctionHemolysis1 moreAcute microcirculatory perfusion disturbances is common in critical illness and associated with higher morbidity and mortality. Recent findings by the investigators' group showed that microcirculatory perfusion is disturbed during cardiac surgery with cardiopulmonary bypass (CPB) and remain disturbed up to 72 (seventy two) hours after surgery. A cardiopulmonary bypass is a machine which takes over heart and lung function, during the procedure. The disturbed microcirculation is associated with organ dysfunction induced by cardiac surgery using CPB, which is frequently seen (up to 42%, forty two percent) and results in a six-fold increase in mortality rate. The underlying cause of disturbed microcirculation is a higher endothelial permeability and vascular leakage and are a consequence of systemic inflammation, hemodilution (dilution of blood), hypothermia and hemolysis (breakdown of red blood cells). To gain the knowledge regarding disturbed microcirculation the investigators previously showed that hemodilution attributes to this disturbed perfusion. Hemodilution lowers colloid oncotic pressure (COP). Also, COP is affected by free hemoglobin, which increases with hemolysis and attributes to a disturbed microcirculation following CPB. This is interesting, as to the best of our knowledge, the effect of minimizing hemodilution and hemolysis during cardiac surgery on the microcirculatory perfusion has never been investigated, but could be the key factor in reducing organ dysfunction.
A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune...
Warm Autoimmune Hemolytic Anemia (wAIHA)The purpose of this study is to evaluate efficacy and safety of ianalumab compared to placebo in patients with warm autoimmune hemolytic anemia, who failed at least one line of treatment.
Hyperhydration in Children With Shiga Toxin-Producing E. Coli Infection
Shiga Toxin-Producing Escherichia Coli (E. Coli) InfectionHemolytic-Uremic SyndromeThe objective of this study is to determine if early high volume intravenous fluid administration (hyperhydration) may be effective in mitigating or preventing complications of shiga toxin-producing E. coli (STEC) infection in children and adolescents when compared with traditional approaches (conservative fluid management).
Sildenafil To Prevent Clot
ThrombosisHemolysisThe advent of continuous flow (CF) pumps for patients with severe heart failure has led to marked improvements in survival; however, pump operation remains fraught with adverse thrombotic events. This climbing rate of thrombosis and stroke during CF pump support has led to a recent warning by the US Food and Drug Administration. Despite a rising incidence of pump thrombosis and its downstream complications of stroke, the hematologic mechanisms behind these devastating adverse events remain uncertain. Recently, it has been recognized that CF pump induced hemolysis precedes and is associated with thrombosis. In-vitro studies show increased platelet function with exposure to products of hemolysis, which is also known to occur in diseases of intravascular hemolysis such as sickle cell anemia. This proposal will investigate if hemolysis associated increased platelet function can be reduced by a potentiation of nitric oxide signaling by an oral phosphodiesterase-5 inhibitor, sildenafil. Elucidating mechanisms of hemolysis induced thrombosis may inform best strategies for prevention of end organ damage and maintaining optimal CF pump operation.
Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia
Warm Autoimmune Hemolytic AnemiaThe main purpose of this study is to evaluate the efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome...
Atypical Hemolytic Uremic SyndromeThe purpose of this Phase 3 study is to determine whether iptacopan (LNP023) is efficacious and safe for the treatment of aHUS in adult patients who are treatment naive to complement inhibitor therapy.
Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic...
Warm Autoimmune Hemolytic Anemia (wAIHA)This is a single group treatment, Phase 2, open-label, study to evaluate the efficacy, safety and pharmacokinetics of rilzabrutinib in adult patients with wAIHA. All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B will continue to receive the study medication for 52 weeks following the Last Patient In (LPI-Part B). There will be a 7-day safety follow-up period after receiving the last dose of study drug either in Part A (for those not eligible for Part B or early terminated) or Part B. The estimated total duration of the study is approximately 137 weeks (Parts A and B), including the follow-up period. For participants deemed ineligible for Part B, the total length of the study will be 29 weeks (Part A only), including screening and the follow-up period. In Part B, participants who temporarily stop rilzabrutinib treatment and maintain a durable response from W50 to W74, will have their EOS visit at Week 75. In this case, participation will be for 79 weeks including the screening period.
A Study of Obexelimab in Patients With Warm Autoimmune Hemolytic Anemia (SApHiAre)
Warm Autoimmune Hemolytic AnemiaThis study aims to examine the efficacy and safety of obexelimab in participants with Warm Autoimmune Hemolytic Anemia (wAIHA).
An Open-label Study of Povetacicept (ALPN-303) in Subjects With Autoimmune Cytopenias
Immune ThrombocytopeniaIdiopathic Thrombocytopenic Purpura2 moreThe goal of this clinical study is to evaluate povetacicept (ALPN-303) in adults with autoimmune cytopenias of immune thrombocytopenia, autoimmune hemolytic anemia, and cold agglutinin disease to determine if povetacicept is safe and potentially beneficial in treating these diseases. During the study treatment period participants will receive povetacicept approximately every 4 weeks for 6 months, with the possibility of participating in a 6-month study treatment extension period.
A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis,...
POEMS SyndromeAmyloidosis2 moreA Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis