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Cross-tumoral Phase 2 clinical trial exploring Crizotinib (PF-02341066) in patients with advanced tumours induced by causal alterations of either ALK or MET.

Primary objective* To study the antitumor activity and safety of crizotinib across predefined tumor types in patients whose tumors are harboring specific alterations in ALK and/or METSecondary objectives* To study the specificity of the kinase…

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…

Establishing an etiological role of the gut microbiome in the antiphospholipid syndrome phenotype

Primary Objective: - to establish if a gut microbiome perturbation affects APS disease phenotype.Secondary Objective(s): - to identify biomarkers that are responsive gut microbiome perturbation.- to establish if gut microbiome perturbation affects…

Flow augmentation study

Primary Objective: The primary objective of this study is to identify whether the Geko system can augment flow compared to IPCS in post-thrombotic limbs before after stenting.(group 1) In group 2 we want to identify whether the Geko with occluded…

GLANZMANN THROMBASTHENIA NATURAL HISTORY STUDY+

The main objective is to determine genetic phenotype and the prevalence of anti-HLA (human leucocyte antigen) and anti-HPA (human platelet antigen) antibodies in patients with Glanzmann thrombasthenia.

Studying extracellular vesicles in immune thrombocytopenia

Recent data shows that patients with ITP have increased levels of the so called platelet-extracellular-vesicles (EVs). In this pilotstudy we would like to characterize these EVs and investigate whether they play a pathogenic role in the maintenance…

Effect of postural change and mobilization on hemoglobin level in patients on the intensive care unit

To measure a change in Hemoglobin levels upon a change in posture from supine to upright in in ICU patients.

RELAX: Effect of EMDR for reduction of pain interference in children with sickle cell disease

The primary objective of this study is to investigate the efficacy of EMDR therapy in reducing pain interference in the life*s of children with SCD. Secondary objectives are to study the efficacy of EMDR in reducing PTSD symptoms, anxiety,…

Safety and efficacy of nadroparin in neonates: an observational study.

Therapeutic nadroparin administration:Primary objective: To develop evidence-based dosing regimens of nadroparin for extremely premature, premature and term neonates by means of the objectification of the PK/PD of nadroparin.Secondary objective: 1)…

Immune monitoring after anti-CD19 CAR T-cel therapy with patients with B-cell NHL

To determine the factors that contribute to response to CAR T-cell therapy, both at the tumour-end, as well as on the T-cell side to understand mechanisms of efficacy and immune escape in order to optimize this treatment and to reduce toxicity

Understanding clot structure in thrombosis and bleeding disorders

The aim of this fundamental research is to optimize methods and to investigate the effects of different variables in the human body on the clot structure, contributing to the knowledge of blood clotting, cardiovascular disease, and bleeding…

A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)

This study has been transitioned to CTIS with ID 2024-512747-23-00 check the CTIS register for the current data. Study AG348-C-018 is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to demonstrate the clinical…

A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other Luspatercept (ACE-536) clinical trials.

This study has been transitioned to CTIS with ID 2022-502498-40-00 check the CTIS register for the current data. Primary Objective: To evaluate the long-term safety (including progression to acute myeloid leukemia (AML) and/or other malignancies/pre…

Oral versus intravenous iron in IBD patients with anti-inflammatory therapy

Regarding the current recommended once daily dosing of oral iron (with probably less side effects than with a 3 times daily dose), and the decrease of hepcidin in patients on immunosuppressive medication, we hypothesize that patients with mild to…

An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase II and Phase III studies with iptacopan

This study has been transitioned to CTIS with ID 2023-509843-28-00 check the CTIS register for the current data. The purpose of this study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to…

A RANDOMIZED, 2-ARM, PHASE 3 STUDY OF ELRANATAMAB (PF-06863135) VERSUS LENALIDOMIDE IN PATIENTS WITH NEWLY DIAGNOSED MULTIPLE MYELOMA AFTER UNDERGOING AUTOLOGOUS STEM-CELL TRANSPLANTATION

This study has been transitioned to CTIS with ID 2023-508897-27-00 check the CTIS register for the current data. Primary:• To compare the efficacy of elranatamab vs lenalidomide Secondary:- To compare the efficacy of elranatamab vs lenalidomide- To…

A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy in subjects with DIPSS - intermediate or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis and previously treated with ruxolitinib

The primary objective of the study is to evaluate the percentage of subjects with at least 35%spleen volume reduction in the fedratinib and the BAT arms.The secondary objectives are:• To evaluate the percentage of subjects with at least 50%…

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.

A 24-week with possible extension, prospective, multicenter, randomized, double blind, placebo-controlled, 2-parallel group with a randomization 1:1, phase III study to compare efficacy and safety of oral masitinib to placebo in treatment of patients with Smouldering or Indolent Severe Systemic mastocytosis with handicap, unresponsive to optimal symptomatic treatment

To evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from Smouldering or Indolent SevereSystemic mastocytosis with handicap unresponsive to optimal symptomatic treatment.

Single-cell analysis of somatic mutations to understand and improve blood formation after hematopoietic stem cell transplantation

We aim to quantify the contribution of individual HSCs to blood (re-)generation in pediatric HSCT recipients and their donors, and to unravel the consequences of transplantation on HSC long-term genomic integrity.