IGIV Study for Chronic ITP Patients Ages 3-70
Idiopathic Thrombocytopenic PurpuraIdiopathic (immune) thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by platelet destruction and thrombocytopenia (peripheral blood platelet count < 150 x 10^9/L). IVIG therapy is useful in patients in whom the platelet count has to be raised either due to bleeding signs, or where bleeding is predicted (e.g., surgery or parturition). The primary goal of treatment is to maintain the platelet count at a hemostatic level. This study will test the safety and efficacy of IGIV3I Grifols 10% in the treatment of patients with chronic ITP.
Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic...
Thrombocytopenic PurpuraThe purpose of this study is to assess the safety and tolerability of AMG 531 (romiplostim), a novel thrombopoiesis-stimulating peptibody, and its effect on platelet counts in adults with immune thrombocytopenic purpura.
Safety Study of AMG 531 in Japanese Subjects With ITP
Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)The purpose of the study is to evaluate the efficacy of starting dose of AMG 531 as measured by platelet counts by cohort dose-escalation design.
Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic...
Acquired Thrombotic Thrombocytopenic Purpura (aTTP)The purpose of this study is to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment of acquired thrombotic thrombocytopenic purpura (aTTP) participants.
Therapy of Adults Affected by Idiopathic Thrombocytopenic Purpura With Dexamethasone
ITPCroticosteroid TherapyIn this prospective, randomized, controlled clinical trial investigators' aim was to compare the efficacy and the adverse effect of 3 therapy cycles of HD-DXM versus conventional treatment with PDN for untreated adult patients with ITP. In this study standardized criteria and definitions were used according to consensus international working group guideline for ITPto compare clinical outcomes of the two corticosteroid treatment regimens and determine the superior regimen as a first line strategy for new primary ITP in adults
The Use of Rituximab in Acute Thrombotic Thrombocytopenic Purpura (TTP)
Thrombotic Thrombocytopenic Purpura (TTP)TTP is a rare and serious blood disorder, characterized by the formation of small clots (micro thrombi) within the circulation and can be fatal. The formation of blood clots occurs primarily in the smaller blood vessels, the arterioles and capillaries, associated with multisystem organ involvement, especially the brain and kidneys. TTP has an incidence of approximately 1-3 people/million of the population/year. TTP is due to a decrease in an enzyme, ADAMTS 13 that is released by cells lining blood vessels (endothelial cells). ADAMTS 13 'cleaves' or breaks down very large von Willebrand Factor (vWF) strands. vWF is used in blood clotting. Deficiency or inhibition of the enzyme, results in release of the ultra large vWF into the circulation. Platelets bind to these ultra large vWF multimers, promoting blood clot formation and platelet consumption (thrombocytopenia). In more then 70% of TTP cases no precipitating cause can be found and the majority of these patients have antibodies against ADAMTS 13. Plasma Exchange (PEX) was introduced in the management of TTP in 1977 and the mortality of TTP patients has since decreased from approximately 90% to 15-20%. PEX is essential in TTP treatment as plasma contains the missing enzyme ADAMTS 13. Rituximab (licensed and internationally used monoclonal antibody) selectively acts on white blood cells known as B-lymphocytes or B cells that produce the antibody to ADAMTS 13. By inhibiting ADAMTS 13 antibody production, ADAMTS 13 activity increases, resulting in remission. Rituximab has been used in our institutions in patients with acute TTP that are refractory to standard treatment - PEX. The resulting remission has been dramatic, with a non-toxic side effect profile and no patients to date has relapsed (longest follow-up 19 months) following Rituximab therapy. Therefore, we plan to use Rituximab with PEX in patients who present with acute TTP.
Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients...
Idiopathic Thrombocytopenic PurpuraThrombocytopenia4 moreThe purpose of this study is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric patients with Immune thrombocytopenia purpura (ITP) as measured by durable platelet response.
Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With...
Acquired Thrombotic Thrombocytopenic PurpuraThis study was a Phase II, single-blind, randomized, placebo-controlled trial to determine whether anti-vWF Nanobody is safe and effective as adjunctive treatment in patients with aTTP. Patients received either placebo or anti-vWF Nanobody as adjunctive therapy to plasma exchange (PE).
Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic...
Immune Thrombocytopenic PurpuraThis trial is designed as a multi-centre, single-dose, exploratory dose-finding, open label trial evaluating the safety and efficacy of Sym001 in 4-9 consecutive cohorts. Subjects will receive a single IV dose of Sym001.
Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia...
ThrombocytopeniaIdiopathic Thrombocytopenic PurpuraThe purpose of this study is to evaluate changes in bone marrow morphology (structure) after long-term exposure to romiplostim.