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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 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…

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…

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,…

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…

An open-label, uncontrolled, multicenter, multinational study on the efficacy and safety of administration of donor lymphocytes depleted of alloreactive T-cells (ATIR), through the use of TH9402 and light treatment in an ex vivo process, in patients receiving a CD34-selected peripheral blood stem cell graft from a related, haploidentical donor.

Primary objective:• To study the effects of the administration of a donor lymphocyte preparation selectively depleted of host alloreactive T cells (ATIR) to patients with hematologic malignancies on 6 months and 12 months transplant related…

Erythrocytapheresis versus Phlebotomy as maintenance therapy in patients with hereditairy hemochromatosis; a randomised, single blinded, sequential, cross-over trial

The primary objective of the study is to determine the gain in effectiveness of TE compared with P when applied to remove excess iron during maintenance therapy of HH patients.

A Phase I Study to Evaluate the Safety and Tolerability of the Histone Deacetylase Inhibitor, CHR-2845, in Patients with Advanced or Treatment Refractory Haematological Diseases or Lymphoid Malignancies

Primary objective:* To determine the safety, tolerability, dose-limiting toxicities (DLT), maximum acceptable dose (MAD) and maximum tolerated dose (MTD) of CHR-2845 when administered orally to patients with advanced or treatment refractory…

Co-infusion of haematopoietic stem cells from a haplo-identical donor and single unit unrelated cord blood in patients with a high risk of relapse:
A Phase l/ll study to assess safety and to investigate the biological mechanism of the anti-tumor response

To study the safety of co-infusion of a alphabetaT-/CD19 B-cell depleted haematopoietic stem cells from haplo-identical donor and a single unit cord blood unit and to investigate the anti-tumor responses from both grafts.

Role of flow cytometry in the assessment of restoration of hematopoiesis during standard Exjade® treatment in patients with low- intermediate I risk myelodysplastisc syndromes, a pilot study

- Primary objective:To evaluate the flow cytometric characteristics of the bone marrow compartment before and during treatment with Exjade® in low and intermediate-I risk myelodysplastic syndromes.- Secondary objectives:To correlate the flow…

Biobank Hematological Stem Cell Transplantation

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Bortezomib in combination with continuous low-dose oral cyclophosphamide and dexamethason followed by maintenance in primary refractory or relapsed bortezomib naïve multiple myeloma patients

Evaluation of the safety and efficacy of bortezomib combined with cyclophosphamide and dexamethasone for induction and maintenance therapy.

Efficacy and safety of canakinumab in Schnitzler syndrome

Primary: To determine whether IL-1 inhibition by Canakinumab is efficacious in treatment of Schnitzler syndrome.Secondary:1. To assess the effect of canakinumab on Schnitzler syndrome (clinical signs/symptoms and inflammatory biomarkers C-reactive…

Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety
Study with PA101 in Patients with Indolent Systemic Mastocytosis

Primary:* To determine the efficacy profile of PA101 delivered via a high efficiency nebulizer (eFlow®,PARI) in comparison with placebo following 6 weeks of treatment in patients with indolentsystemic mastocytosis (ISM) who are symptomatic despite…

A multicenter study of apheresis collection of peripheral blood mononuclear cells (PBMC) in patients with CD19 expressing malignancies who could be eligible for a CTL019 clinical research trial

The rationale for this protocol is to use standardized procedures to obtain PBMCs from adult patients with a diagnosis of a CD19 expressingmalignancy for which a CTL019 treatment protocol is currently enrolling or under IRB/EC review, and to…

Characterization of buffy-coat-derived granulocytes for clinical use: - identifying clinical and laboratory parameters for decision making

• To find surrogate markers in plasma in order to identify patients, which may benefit from granulocyte transfusions and which are at risk for transfusion complications.• To determine - in combination with the outcomes of the NEPTUNIS study -…

A Phase 2, Multicohort Study of Daratumumab-Based Therapies in Participants with
Amyloid Light Chain (AL) Amyloidosis

This study has been transitioned to CTIS with ID 2023-507069-25-00 check the CTIS register for the current data. The primary objectives for the study are: Cohort 1, to characterize cardiac safety of different Daratumumab, cyclophosphamide,…

The pharmacokinetics and pharmacodynamics of eculizumab in patients with paroxysmal nocturnal hemoglobinuria.

To describe the real-world population pharmacokinetics and pharmacodynamics of eculizumab in unselected PNH patients

A phase 3b, prospective, open-label, uncontrolled, multicenter study on long-term safety and efficacy of rVWF in pediatric and adult subjects with severe von Willebrand disease (VWD)

To evaluate the efficacy of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in adult and pediatric / adolescent subjects (aged 12 to <18 years) during the…