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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…

Randomized, open label, multicenter phase III study of Efficacy and Safety in POlycythemia vera subjects who are resistant to or intolerant of hydroxyurea: JAK iNhibitor INC424 tablets verSus bEst available care

Earlier research nas shown the emerging efficacy and safety profile for INC424, which supports further studies in PV subjects who demonstrate resistance or intolerance to HU therapy [Barosi et al.2009]. This pivotal phase III trial (CINC424B2301) is…

Recovery and survival of platelet concentrates in plasma and in 3 additive solutions

The objectives of this study is to determine the recovery and survival of platelet concentrates; in the first phase, a comparison will be made for platelet concentrates in plasma stored for 2-3 days versus those stored for 6-7 days; in the second…

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 3, Randomized, Open-Label, Active-Controlled Study to Evaluate the Efficacy and Safety of roxadustat in the Treatment of Anemia in Chronic Kidney Disease Patients Not on Dialysis

The primary objective of this study is to evaluate the efficacy of roxadustat compared todarbepoetin alfa in the treatment of anemia in non-dialysis dependent Chronic Kidney Disease (NDD-CKD)subjects.The secondary objectives of this study are to:*…

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

A Phase 1, Prospective, Open Label, Two Period, Fixed Sequence, Dose-Escalation Study of the PK and Safety of BAX 826 (PSA-rFVIII) in Previously Treated Patients with Severe (FVIII <1%) Hemophilia A: Protocol number: 291501

1. To assess tolerability and safety of BAX 826 after a single infusion in previously PTPs with severe hemophilia A.2. To determine the pharmacokinetic (PK) parameters of BAX 826 compared to ADVATE.3. To evaluate the impact of anti- PSA antibodies…

Improving quality of survivorship for breast cancer-related lymphedema by lymphatic microsurgery: A randomized controlled trial

To assess the effectivity of LVA on arm volume in comparison to standard therapy after 12 months.

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.

A Phase 3 clinical study to determine the pharmacokinetics, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in subjects diagnosed with von Willebrand disease

Primary objective:• Assess the pharmacokinetics (PK) of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary VWDSecundary objectives:• Compare the…

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,…