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An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects with Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies

Objectives: Primary:• To evaluate the long-term safety and tolerability of AG-348Secondary:• To evaluate the long-term efficacy of AG-348• To evaluate the efficacy of AG-348 in increasing hemoglobin (Hb) concentrations in subjects who previously…

A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITH COMPLEMENT INHIBITORS

This study has been transitioned to CTIS with ID 2023-506498-36-00 check the CTIS register for the current data. To evaluate the efficacy of crovalimab compared to eculizumab

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.

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.

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.

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…

A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy in subjects with DIPSS - intermediate or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis and previously treated with ruxolitinib

The primary objective of the study is to evaluate the percentage of subjects with at least 35%spleen volume reduction in the fedratinib and the BAT arms.The secondary objectives are:• To evaluate the percentage of subjects with at least 50%…

A Phase 2, Open-label, Dose Escalation and Dose Expansion Study of KER-047 for the Treatment of IRIDA

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…

Objective Neurocognitive assessment of young children with sickle cell disease by eye-tracking

The main aim is to study early neurocognitive functioning and development in very young children with SCD. The secondary aims are to identify determinants (risk and protective factors) and biomarkers of early neurocognitive deficits in SCD and to…

A Phase 1 Study to Assess the Mass Balance, Excretion, and Pharmacokinetics of [14C]-GBT021601, an Oral Hemoglobin S Polymerization Inhibitor, in Healthy Participants

In this study we will investigate how quickly and to what extent GBT021601 is absorbed, transported, and eliminated from the body. For this study, GBT021601 is radioactively labelled with carbon-14 (14C). In this way, GBT021601 can be traced in…

A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS

This study has been transitioned to CTIS with ID 2023-506526-37-00 check the CTIS register for the current data. To evaluate the safety and tolerability of crovalimab compared witheculizumab

An Open-Label Study to Evaluate the Efficacy and Safety of AG-348 in Regularly Transfused Adult Subjects With Pyruvate Kinase (PK) Deficiency

The primary objective of the study is to evaluate the efficacy of treatment with AG-348, as assessed by the reduction in transfusion burden.Secondary: The secondary objective of this study is to evaluate the safety of treatment with AG 348.

A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently treated with Eculizumab

Efficacy of ALXN1210Sub-study: to Evaluate Patient Preference for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Investigating the interplay between iron (overload) and erythropoiesis in rare hereditary anemias.

Investigate regulators of (disturbed) iron homeostasis and cellular determinants of iron overload in RHA. to decipher crucial elements of the crosstalk between iron metabolism and erythropoiesis

Remote patient Monitoring in adults receiving red blood cell Transfusion for hematological disorders * a Pilot study of the physIQ Real World Monitoring platform using wearable biosensors

Primary objectives:- to explore transfusion induced differences in vital (activity) parameters such as heart rate, respiratory rate and activity before and after RBC transfusions in regularly transfused patients using the pinpointIQ* system.- To…

The Effect of Crizanlizumab on Cerebral Perfusion and Oxygenation in Sickle Cell Patients (CSEG101ANL01T)

To study the effect of crizanlizumab on the hemodynamics of the cerebral vasculature (CBF and CVR)

An In Vivo 24-Hour Recovery Study of Leukoreduced RBCs After Automated Separation of Whole Blood by the Reveos System and Storage With Non-DEHP Disposables.

To evaluate whether the RBCs derived from WB and processed with the Reveos system using the non-DEHP disposable sets meet the US criteria for 24-hour recovery after 42 days of storage.

:Improve the outcome of Preterm born neonates by Cord Blood Transfusions

Primary objective: To assess the feasibility of donor screening, collection, production and storage of sufficient CB-RBC products to potentially meet the transfusion need in extremely preterm neonates.