Bortezomib to Treat Significant Complication of HSCT
Allogeneic Stem Cell TransplantationRefractory Autoimmune Cytopenia(s)The purpose of this trial is to study the safety and effectiveness of a drug called Bortezomib for the treatment of low blood cell counts after bone marrow transplant.
Safety and Efficacy Study of Lusutrombopag for Thrombocytopenia in Patients With Chronic Liver Disease...
Chronic Liver DiseaseThrombocytopeniaThe primary purpose of this study is to compare the efficacy of lusutrombopag with placebo for the treatment of thrombocytopenia in patients with chronic liver disease who are undergoing elective invasive procedures.
Daily Prednisone Versus Pulsed Dexamethasone in Treatment-naïve Adult Patients With Immune Thrombocytopenia...
PurpuraThrombocytopenic1 morePatients above age 18 with a first episode of immune thrombocytopenia are randomized 1:1 between 2-4 weeks of daily prednisone (1 mg/kg/d) with subsequent dose tapering (arm A) and six 3-week cycles of pulsed dexamethasone (0.6 mg/kg/d, days 1-4; arm B). The primary endpoint is duration of remission defined as platelets ≥50/nl.
Validation of a Predictive Model of Response to Romiplostim in Patients With IPSS Low or Intermediate-1...
Myelodysplastic SyndromesThere are currently no licensed drugs in the EU to treat thrombocytopenia in MDS patients classified as IPSS low/int-1. Prior studies with romiplostim (a TPO receptor agonist) in MDS found that baseline concentration of TPO as well as transfusion history were predictive of subsequent response in a retrospective model. The current prospective study has the aim to explore whether both pretreatment variables (endogenous TPO, TPO-level, platelet transfusion history) can predict the response to subsequent short-term treatment with romiplostim.
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once-daily Oral Avatrombopag in...
Thrombocytopenia Associated With Chronic Liver DiseaseThis is a phase 2, randomized, double-blind, placebo-controlled, parallel group study using avatrombopag for Japanese subjects with thrombocytopenia associated with chronic liver disease. This study will assess the effect of avatrombopag on platelet counts in Japanese subjects. Subjects will be enrolled into 2 cohorts according to the mean platelet count measured at Screening and Baseline. Within the lower baseline platelet count cohort (less than 40 x 10^9/L), subjects will be randomized in a 1:1:1:3 ratio to receive placebo, 20 mg avatrombopag, 40 mg avatrombopag, or 60 mg avatrombopag for 5 days. Within the higher baseline platelet count cohort (from 40 to less than 50 x 10^9/L), subjects will be randomized in a 2:1:2 ratio to receive placebo, 20 mg avatrombopag, or 40 mg avatrombopag for 5 days.
Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia
Primacy Immune ThrombocytopeniaA clinical study to evaluate the efficacy of a drug called eltrombopag in adult patients affected of primary immune thrombocytopenia as first treatment to address the disease.
Immunomodulation With Romiplostim in Young Adults With ITP
Immune ThrombocytopeniaThe study aims to investigate immunomodulatory effects of thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it. It is hypothesized that the early phase of this autoimmune disease may exhibit a stronger immunomodulatory potential in response to a stimulus, such as romiplostim. Such a process may subsequently be capable to induce regulatory mechanisms or tolerance. Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC).
An Investigation of rhTPO With Different Frequencies in the Management of ITP
Immune ThrombocytopeniaThe project was undertaking by Qilu Hospital of Shandong University and other well-known hospitals in China. In order to study the efficacy and safety of different dose and frequency Recombinant Human thrombopoietin in treating the primary immune thrombocytopenia (ITP)
Human Placental-Derived Stem Cell Transplantation
Mucopolysaccharidosis IMucopolysaccharidosis VI14 moreThe purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.
Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder...
Idiopathic Thrombocytopenic PurpuraThe purpose of this study is to investigate the efficacy, safety and tolerability of eltrombopag in children with previously treated chronic immune thrombocytopenia who are between 1 and 17 years of age. This is a 2 part study. In part 1, patients will be randomized to receive either eltrombopag or placebo for 13 weeks. All patients who complete part 1 will enter part 2. In part 2, all patients will receive 24 weeks of eltrombopag.