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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),…

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…

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…

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,…

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 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…

A RANDOMIZED DOUBLE-BLIND PHASE IIA STUDY EVALUATING THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF CROVALIMAB AS ADJUNCT TREATMENT IN PREVENTION OF VASO-OCCLUSIVE EPISODES (VOE) IN SICKLE CELL DISEASE (SCD)

This study has been transitioned to CTIS with ID 2022-502542-28-00 check the CTIS register for the current data. This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of crovalimab compared with placebo as adjunct…

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)

A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients with Pyruvate Kinase Deficiency

CORE PERIODPrimary:* Evaluate the safety and tolerability of up to 24 weeks of AG-348 administration in patients with pyruvate kinase deficiency (PK deficiency).Secondary:* Evaluate the pharmacokinetics (PK) of AG-348 and the metabolite AGI-8702.*…

A multi-centre, phase II, randomized, double-blind, placebo-controlled study to investigate efficacy and safety of sevuparin infusion for the management of acute vaso-occlusive crisis (VOC) in subjects with sickle-cell disease (SCD)

Primary objective:The primary objective of this study is to assess the time to painful VOC resolution, measured from the first dose of sevuparine given to achievement of crises resolution, as compared to placebo. Secondary objectives: The secondary…