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Prospective Follow-up Study for Patients who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)

This is a follow-up (FU) study for subjects who completed Study ALX0681-C301 (HERCULES) according to the protocol (i.e., completed the Final [28 day] FU visit). Subjects who completed Study ALX0681-C301 will be given the option to participate in…

Combined Molecular Microscopy for Therapy and Personalized Medication in Rare Anaemias Treatments

(i) identification and cellular characteristics of red blood cells from patients with altered ion homeostasis and delivery of novel and easy-to-use diagnostics for rare anemia*s, for patients with: a) a known primary defect in the ion channels of…

A Phase III double-blind, randomized, parallel group, multicenter placebo-controlled trial to study the efficacy and safety of caplacizumab in patients with acquired thrombotic thrombocytopenic purpura.

Currently, caplacizumab is being developed for treatment of acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare and potentially life-threatening thrombotic microangiopathy, in which accumulation of ULvWF multimers leads to an increased…

Disturbed ion homeostasis in hereditary hemolytic anaemia

Primary objectives:(i) To study the pathophysiology, in particular the role of disturbed ion channel function in hereditary haemolytic anaemia(ii) To define novel intra- and extracellular (bio)markers in hereditary haemolytic anaemia. Secondary…

Correcting mutations in vitro using CRISPR-Cas9; towards autologous stem cell transplantation in sickle cell disease and X-linked severe combined immunodeficiency

Preparation for in vivo correction of SCD and X-SCID causing mutations by CRISPR-Cas9 by in vitro studies in cell lines

A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Long-term Safety and Efficacy of Darbepoetin Alfa Administered at 500 µg Once-Every-3-Weeks (Q3W) in Anemic Subjects With Advanced Stage Non-small Cell Lung Cancer Receiving Multi-cycle Chemotherapy.

The purpose of this study is to evaluate the safety of darbepoetin alfa, inclusive of the effects on survival and cancer progression, and the necessity of blood transfusions.

The effects of red blood cell transfusion on the microcirculation in chronic anemic preterm neonates.

To determine the effect of a single red blood cell transfusion on the microcirculation.

A Phase 2, Randomized, Double-blind, Placebo-controlled, Multicenter Study Comparing Siltuximab Plus Best Supportive Care to Placebo Plus Best Supportive Care in Anemic Subjects with International Prognostic Scoring System Low- or Intermediate-1-Risk Myelodysplastic Syndrome

The primary objective is to assess the clinical efficacy of siltuximab (a chimeric (murine-human) IgG1* mAb that specifically binds human IL-6 with high affinity and prevents its interaction with the IL-6 receptor, glycoprotein (GP) 80),…

PHASE I, DOUBLE BLIND, PLACEBO-CONTROLLED, DOSE-ESCALATION STUDY TO ASSESS THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS OF FMX-8 IN HEALTHY MALE VOLUNTEERS

Primary:To determine the safety, tolerability, and pharmacokinetic (PK) profile of single ascending intravenous (iv) doses and of multiple ascending iv doses of FMX-8 in healthy subjectsSecondary:To evaluate the pharmacodynamics (PD) of FMX-8 in…

Central Hemodynamics, Augmentation index and Microcirculation after Phlebotomy

Our principal aim is to assess the effects of phlebotomy on aortic augmentation (AIx) and central BP in supine position and after standing; second, to assess the effects of counter maneuvers (leg crossing and thigh compression) on aortic…

Determining the role of Von Willebrand factor and ADAMTS13 in the adhesion of sickled erythrocytes to endothelial cells

To determine the role of VWF and ADAMTS13 in the adherence of sickled erythrocytes to vascular endothelium.

The efficacy of blood transfusions to improve microcirculatory oxygen delivery in anemic patients.

The main purpose of this project is to investigate the conditions under which blood transfusions are successful in promoting microcirculatory circulation and oxygenation in anemic patients. In this project we wish to apply our state of the art…

Protocol C: Microcirculatory response to phlebotomy in adult patients with Eisenmenger syndrome.

Aim of the proposed study will be to investigate whether microvascular blood flow at baseline is impaired in Eisenmenger patients who experience symptoms of the *hyperviscosity syndrome*. Furthermore, in view of the controversy whether to perform…

An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)

The purpose of Part 1A is to investigate how safe RO7112689 is and how well RO7112689 is tolerated. Part 1A will also investigate how quickly and to what extent RO7112689 is absorbed into, distributed in, and eliminated from the body (this is called…

An open-label extension study to evaluate the long-term safety,
tolerability, and efficacy of REGN3918 in patients with paroxysmal
nocturnal hemoglobinuria

The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis (ie, proportion ofpatients achieving lactate dehydrogenase (LDH) * 1.5× upper limit of normal (ULN) over 26 weeks) of…

An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Received Complement Inhibitor Therapy

The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patientswith active PNH who are treatment-naive to complement inhibitor therapy or have not recently received…

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics and
Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe
Hemolytic Disease of the Fetus and Newborn (HDFN)

Primary Objectives: - To evaluate the safety in mother and neonate/infant of nipocalimab administered to pregnant women at high risk for EOS-HDFN. - To evaluate the efficacy of nipocalimab as measured by proportion of patients with live birth at or…

The Effect of Voxelotor on Cerebral Perfusion and Oxygenation (ESR-C005)

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

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, multicenter, randomized, double-blind study to assess efficacy and safety of two doses of crizanlizumab versus placebo, with or without hydroxyureal/hydroxycarbamide therapy, in adolescent and adult sickle cell disease patients with vaso-occlusive crises (STAND)

This study has been transitioned to CTIS with ID 2023-508689-14-00 check the CTIS register for the current data. To compare the efficacy of 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of VOC leading to healthcare visit, in…