Search for a trial

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…

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…

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.

A randomized, 2 part, 4-treatment, 2-way cross-over study in healthy volunteers to compare the pharmacokinetic and pharmacodynamics profiles of 1 *g/kg of DA-3880 and EU sourced AND EU Aranesp® (AMGEN) after single intravenous or subcutaneous administration

The purpose of the study is to investigate to what extent DA-3880 is absorbed and eliminated from the body (this is called pharmacokinetics) as compared to the above mentioned Aranesp® formulation. In addition, the effect DA-3880 on the number of…

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…

Perineal block with prilocaine during labour: effect on the newborn infant. A pilot study.

To determine the rate of transmission of prilocaine across the placentaTo determine the levels of methemoglobinemia in newborns after perineal block with prilocaine during labor (following the regimen used in our hospital)To determine the levels of…

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…

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

The primary objective of this study is to assess the noninferiority of ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor. Noninferiority will be claimed if after 26 weeks of treatment…

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)

A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab

Primary:• To evaluate PK noninferiority of ravulizumab SC versus ravulizumab IV in adult patients with PNH Secondary• To characterize PK of ravulizumab SC• To characterize PD of ravulizumab SC• To characterize immunogenicity of ravulizumab SC• To…

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…

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

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