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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…

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.

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…

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…

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…

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,…

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)

To evaluate the efficacy of danicopan as compared to placebo as add-on therapy to a C5 inhibitor at 12 weeks.

A PHASE IB, RANDOMIZED, PLACEBO-CONTROLLED STUDY EVALUATING THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF CROVALIMAB FOR THE MANAGEMENT OF ACUTE UNCOMPLICATED VASO*OCCLUSIVE EPISODES (VOE) IN PATIENTS WITH SICKLE CELL DISEASE (SCD)

This study has been transitioned to CTIS with ID 2022-502546-26-00 check the CTIS register for the current data. This study will evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of crovalimab compared with placebo for the…

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…

'A phase 1 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-dose intravenous and subcutaneous administration and multiple-dose subcutaneous administration of OMS906 in healthy subjects'

The purpose of this study is to investigate how safe the new compound OMS906 is and how well it is tolerated when it is administered to healthy volunteers. OMS906 has not been administered to humans before. It has been previously tested in the…

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…