Evaluating the Pharmacokinetics and Pharmacodynamics of Romiplostim in Patients With Immune Thrombocytopenia...
Immune Thrombocytopenia (ITP)The objective of evaluating the pharmacokinetics and pharmacodynamics of romiplostim in patients with immune thrombocytopenia.
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study...
Primary Immune ThrombocytopeniaThe purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity...
Autoimmune Lymphoproliferative SyndromeAutoimmune Cytopenia19 moreThe main objective of this study is to generate diagnosis and therapeutic-decision tools through the identification of molecular causes of PIDs with autoimmunity/inflammation and the variability in disease outcome at the transcriptional level using a combination of omics signatures (transcriptomics, epigenomics, proteomics, metagenomics, metabolomics and lipidomics).
The Combination of ATRA and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia...
Immune ThrombocytopeniaRandomized, open-label, multicenter study to compare the efficacy and safety of ATRA plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
Evaluating the Efficacy and Safety of Different Human Thrombopoietin (rhTPO) Regimens in the Treatment...
ITPThis is a review of the efficacy and safety of different recombinant human thrombopoietin (rhTPO) regimens in the treatment of patients with primary immune thrombocytopenia (ITP).
Evaluation of the Effect in Platelet Count of Atorvastatin and N-acetyl Cysteine
Primary Immune ThrombocytopeniaThe purpose of this study is to assess the ability of Atorvastatin and N-acetyl cysteine to elevate the blood (serum) platelet count in patients with Immune Thrombocytopenia as a mean of a new treatment.
Efficacy of Sialic Acid GNE Related Thrombocytopenia
ThrombocytopeniaSialic Acid-Extended Release (SA-ER, aceneuramic acid, UX001) is an extended release formulation of sialic acid (SA, also known as N-acetylneuraminic acid or NANA). The SA-ER is currently studied as a substrate replacement therapy for patients with GNE myopathy. The investigators plan to study the SA-ER compound in a cohort of five patients with GNE-related thrombocytopenia.
Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received...
Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)This is a Phase I/II multicenter study which aims to assess the toxicity profile of Romiplostim in patients with transfusion-dependent thrombocytopenia after allogeneic HSCT. A total of 24 patients with transfusion-dependent thrombocytopenia after allogeneic HSCT will be included. The main endpoint is the incidence and severity of adverse events.
An Investigation of Dexamethasone With Different Doses in the Management of Immune Thrombocytopenia...
Immune ThrombocytopeniaThe project was undertaking by Qilu Hospital of Shandong University and other 7 well-known hospitals in China. In order to report the efficacy and safety of different dose dexamethasone in treating the immune thrombocytopenia (ITP).
Evaluation of the Efficacy of Platelets Treated With Pathogen Reduction Process
Aplasia With Expected ThrombocytopeniaThis study is a multicentre, double-blind, randomized therapeutic trial. The primary objective of this study is to evaluate non-inferiority with regard to prevention and control of haemorrhage: of platelet concentrates treated by pathogen reduction(Intercept amotosalen and UVA procedure) compared with the usual platelet concentrates (in additive solution intersol), reference arm, and compared with platelet concentrates re-suspended in autologous plasma (historic arm) These three products are available and authorised by ANSM (formerly AFSSAPS). The secondary objectives is to evaluate the transfusion needs, transfusion outcomes and safety and the decreased frequency of grade 2 or higher side effects related to transfusion allergy to platelets.