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Response to DDAVP In mild Hemophilia A patients, in Search for dEterminants

The aim of this project is to investigate the association between clinical and genetic factors and DDAVP response in MHA patients and to determine their relative contributions to the DDAVP response.

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 Ib, multi-center, open-label, dose-escalation study of PIM447 in combination with ruxolitinib (INC424) and LEE011 administered orally in patients with myelofibrosis (CPIM447X2104C)

Primary: To estimate the MTD and/or RDE for each of the following three treatment arms in patients with myelofibrosis.* PIM447 plus ruxolitinib (doublet)* Ribociclib (LEE011) plus ruxolitinib (doublet)* PIM447 plus ruxolitinib and Ribociclib (LEE011…

A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura.

• The primary objective of this study is to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP.• The secondary objectives of this study are to establish the long-term efficacy of fostamatinib in achieving and…

A phase I, multicenter, open-label study of oral LGH447 in patients with acute myeloid leukemia or high risk myelodysplastic syndrome (CLGH447X2102)

Primary: To determine the MTD and/or RDE of LGH447 with or without midostaurin. Secondary: 1. To characterize the safety and tolerability of LGH447 with or without midostaurin at the MTD and/or RDE.2. To assess any observed antitumor activity of…

Efficacy and Safety of NNC 0129-0000-1003 during Surgical Procedures in Patients with Haemophilia A

Primary Objective: * To evaluate the haemostatic effect of N8-GP during surgical procedures in patients with haemophilia A. Secondary Objectives * To evaluate the general safety including immunogenicity of N8-GP when used for prevention and…

7-day study of the safety, efficacy and the pharmacokinetic and pharmacodynamic properties
of oral rivaroxaban in children from birth to less than 6 months with arterial or venous
thrombosis

The primary objective is:- to characterize the pharmacokinetic/pharmacodynamic profile of a 7-day treatment with oral rivaroxabanThe secondary objectives are:- to assess the incidence of major bleeding and clinically relevant non-major bleeding- to…

30-day, single-arm study of the safety, efficacy and the pharmacokinetic and pharmacodynamic properties of oral rivaroxaban in children with various manifestations of venous thrombosis.

-Assess the incidence of major bleeding and clinically relevant non-major bleeding -Assess the incidence of recurrent venous thromboembolism -Characterize the pharmacokinetic/ pharmacodynamic profile of a 30-day treatment with oral rivaroxaban

A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis

Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.

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

Umbilical cord blood transplantation in high-risk hematological patients using stemregenin-1 expanded hematopoietic stem cells.
A feasibility study focussing on engraftment and hematopoietic recovery.

To study the feasibility of single UCBT with one ex-vivo SR-1 expanded unitTo assess side effects and TRM after single UCBT with one expanded unitTo assess engraftment and engraftment kinetics; to evaluate immune reconstitution, acute and chronic…

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…

Study 200170: A Rollover Study to Provide Continued Treatment with Eltrombopag

To provide continuing treatment with eltrombopag for subjects who are currently participating in a GSK sponsored investigational study of eltrombopag (parent study) and to collect long term safety data.

Double umbilical cord blood transplantation in high-risk haematological patients.
A phase II study focussing on the mechanism of graft predominance

Objective of the study is to evaluate whether parameters can be identified that predict which graft ultimately prevails following cord blood transplantation after a reduced intensity conditioning regimen in adult patients .In addition engraftment,…

intra-individual platelet activation, as meased by PFA-200, at different time lapses after aspirin intake

To eveluate the effect of amount of time past after last aspirin intake on platelet activability

Bloodloss study after Aortic valve replacement
Impact of Mecc perfusion vs conventional ECCperfusion

Peri-operative Bloodloss after Aortic valve replacement

Effect of three B-cell inhibitors: rituximab, alemtuzumab and bortezomib on anti-A and/or anti-B titer kinetics.

The goal of this study is to find out which B-cell inhibitor results in the best reduction of anti A/B antibody levels at which time interval, to make the best choice for a particcular drug in the desensitization treatment in patients receiving an…

MAGNITUDE-A Phase 3 Evaluation of the Safety and Efficacy of Lambda/RBV/DCV in Treatment Naïve Subjects with Chronic HCV Infection, who have Underlying Mild or Moderate Hemophilia or Patients who are Prior Relapsers to Pegylated interferon alfa/RBV;Revised Protocol 01 Incorporates Amendment 02 and Administrative Letter 01 and 02;+ Pharmacogenetics Blood Sample Amendment Number 01 - Site Specific (version 1.0, dated 11-Sep-12)

Primary Objectives:The primary objective for this study is to evaluate the proportion of subjects who achieve SVR12 (HCV RNA < LLOQ (target not detected) at post-treatment follow-up Week 12 in subjects with GT-1b, -4 and GT-2, -3.Secondary…

An Open-Label Study of the Safety and Efficacy of ReFacto AF in Previously Untreated Patients in Usual Care Settings

The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients (no prior exposure to factor products or any blood products) of less than 6 years of age.

Assessing fetal hemoglobin in patients on Tranylcypromine

The objective of ths study is to investigate whether use of Tranylcypromine, a monoamine oxidase inhibitor, results in increased expression of HbF in adults. If this is the case, we will use the results of this study to design follow-up research…