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Perioperative Management of Patients who are Receiving a Novel Oral Anticoagulant (Dabigatran, Rivaroxaban or Apixaban) and Require an Elective Surgery or Medical Procedure: A Prospective Cohort Study (PAUSE)*

1.1 Overall Aim: To establish a safe, standardized protocol for the perioperative management of patients with atrial fibrillation (AF) who are receiving a novel oral anticoagulant (NOAC) drug, either dabigatran, rivaroxaban or apixaban, and require…

Disease activity and iron status in children with inflammatory bowel disease

Primary Objective: To investigate the relationship between disease activity and iron deficiency (i.e. absolute and functional ID combined) in children with IBD. Secondary Objectives: 1.To establish the prevalence of iron deficiency (both absolute…

Sports Participation and Injuries in Patients with Haemophilia

Primary objectives:1. To quantify physical activity, sports participation and exposure according to age, haemophilia severity and joint status;2. To assess the incidence of sports-related injuries in those who participate in sports at least once…

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 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…

A Phase 1/2 Study of CPI-0610, a Small Molecule Inhibitor of BET Proteins: Phase 1 (Dose Escalation of CPI-0610 in Patients with Hematological Malignancies) and Phase 2 (Dose Expansion of CPI-0610 with and without Ruxolitinib in Patients with Myeloproliferative Neoplasms)

Phase 2 (MF Expansion-Prior JAKi Arm 1 and Add-on to JAKi Arm 2) Primary Objectives:- To evaluate splenic response rate by imaging after 24 weeks of treatment in Cohorts 1B and 2B (i.e., in non-TD cohorts)- To evaluate the rate of conversion from…

An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants with Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

The objective of this open-label extension (OLE) study is to assess the effect of long-term safety and treatment effect of Voxelotor in participants who have completed treatment in Study GBT440-031, using the following parameters:- Safety based upon…

Cross-sectional study for evaluation of Platelet function and Immune profile in patients with Chronic ITP

To assess platelet function and coagulation parameters in adult chronic ITP patients in relation to their bleeding phenotype

A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy

This study has been transitioned to CTIS with ID 2024-512585-34-00 check the CTIS register for the current data. Primary: Part 1 • To determine the RP2D of avapritinib in patients with ISM for use in Part 2 and Part 3 of the study.Part 2 • To…

A Phase 3, Open -label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) versus Epoetin alfa for the Treatment of Anemia due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in ESA Naïve Subjects who require Red Blood Cell Transfusions.

This study has been transitioned to CTIS with ID 2022-501485-22-00 check the CTIS register for the current data. Primary objective:• To evaluate the efficacy of luspatercept on RBC transfusion independence (RBC-TI for 12 weeks [84 days] with an…

Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B

This study has been transitioned to CTIS with ID 2024-510738-42-00 check the CTIS register for the current data. The primary aim of the trial is to demonstrate the non-inferiority of CSL222 (formerly AMT-061) (2 × 1013 gc/kg) during the 52 weeks…

Von Willebrand disease in the Netherlands - Prospective study (WiN-Pro)

Our primary aim is to investigate the current bleeding tendency of patients with VWD.

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…

An Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients with Advanced Systemic Mastocytosis

Primary: • The primary objective is to determine adjudicated ORR (CR/CR with partial recovery of peripheral blood counts [CRh] + PR + CI) based on modified IWG-MRT-ECNM consensus response criteria in patients with AdvSM treated with avapritinib and…

A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A

This study has been transitioned to CTIS with ID 2023-505827-29-00 check the CTIS register for the current data. The purpose of this first in human study is to determine if the gene transfer study product, BAY 2599023 (DTX201), is safe and has…

Exploring the clinical utility of a novel multichannel platelet function test in patients with stable coronary disease

The main objective of the current study is to investigate platelet reactivity and underlying mechanisms using the novel multi-microspot analysis in a group of patients with SIHD and a healthy control group.

The BITE study - Bleeding In Thrombocytopenia Explained: A nationwide epidemiological and laboratory case cohort study investigating risk factors for bleeding in hemato-oncology patients

- Identify hemato-oncology patients and conditions with a high versus a low bleeding risk, by epidemiological research and a short questionnaire. - Investigate the association of bleeding related biomarkers with bleeding.

Improving the quality of life of patients with breast cancer-related lymphedema by lymphaticovenous anastomosis (LVA): A randomized controlled trial

The primary objective of this randomized controlled trial is to study the effectiveness of lymphaticovenous anastomosis (LVA) compared to complex decongestive therapy (CDT) in terms of health-related quality of life (HRQoL). Secondary objectives are…

A 4-part Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of M254 in healthy volunteers and in patients with immune thrombocytopenic purpura.

The purpose of this study is to investigate how safe the new compound M254 is and how well it is tolerated when it is administered to healthy volunteers and patients with immune thrombocytopenia (ITP). M254 has not been administered to humans before…

Proton pump inhibition for secondary hemochromatosis in hereditary anemia, a phase III placebo controlled randomized cross-over clinical trial.

Primary Objective to show that PPIs compared to placebo are an effective treatment of secondary hemochromatosis in a relative large number of patients with hereditary anemia and mild iron overload. Secondary Objectives: To assess the safety and side…